The pandemic proved new therapeutics and vaccines are able to navigate through the bureaucratic quagmire when there is the public will to make it happen. New antivirals therapies, monoclonal antibodies, and mRNA vaccines are now saving lives. 

These success stories are outliers. In reality, pharmaceutical and medical device innovations move through the system at a snail’s pace. 

A product must pass through a lengthy FDA approval process to verify safety and efficacy. Despite FDA approval, a new medical device still can not get into the hands of surgeons. New technologies run into a brick wall as they fight for acceptance as an approved product by third-party payers. 

Insurance companies hide behind the word “investigational” to justify the denial of care. An FDA-approved medical device has little value to doctors and patients if none of the third-party payers pay for its use. 

Every insurance company must accept and authorize a new medication or medical device into their approved formulary before it is considered a covered benefit. Even after CMS approval, each managed care Medicare and Medicaid plan also submit new products to this lengthy process.

Medical practices spend countless hours jumping through pre-authorization hoops and appealing denied claims. 

We invest this time and energy to get our patients the most effective options, but patient care does not have to be so hard. Patients want doctors to spend time in the exam room, not writing insurance appeal letters. 

Finn Partners manager of global health, Gil Bashe, summarizes the barriers stifling medical device innovation in his article “Investigational” Is Payer Code for “Coverage Denied”. 

It is time for insurance companies to tear down the walls, stop playing games and help doctors provide the best care to their patients. 

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AMSTERDAM/PRNewswire/ — StrokeViewer enables physicians to provide every stroke patient with the right treatment, as fast as possible. Two million brain cells die every minute until blood flow is restored (Saver, 2006), starting treatment earlier can make the difference between recovery or life-long disability.

Date of Release: Nov. 26, 2020 

This week the FDA approved a 510k application (FDA K200873) for StrokeViewer LVO, an artificial intelligence algorithm for fast triaging of stroke patients. StrokeViewer LVO detects image characteristics associated with a Large Vessel Occlusion (LVO) and alerts physicians. The FDA application was supported by a multi-center clinical study where the performance of the algorithm was retrospectively evaluated in 384 patients from multiple US stroke centers. An expert panel assessed the data to identify LVOs (ICA, M1 and M2) and comparison with the algorithm showed results exceeding the performance goal.

StrokeViewer was developed by the Dutch medtech company NICO.LAB. The cloud-based solution uses artificial intelligence to support physicians in the emergency stroke setting. The FDA-cleared LVO algorithm is embedded in a cloud-based system that sends a notification to the medical specialists involved just minutes after a stroke patient arrives in the hospital. The physicians are able to use their smartphones, in the hospital but also at home, to inspect the CT images in a web viewer and diagnose stroke.

“Medical specialists are under enormous pressure to make fast decisions day and night, but it’s not easy. Complicated assessment and interhospital communication sadly make life difficult for physicians motivated to treat stroke victims effectively” – said Merel Boers, CEO and co-founder of NICO.LAB. “With our first FDA clearance we are now able to show U.S. physicians how impactful the combination of human and artificial intelligence is. And yes, more will follow as we are fully committed to unlocking the full healthcare potential for every patient.”

StrokeViewer is currently in use in Australia and Europe where it has proven to reduce the time from hospital arrival to start treatment for patients with acute stroke, leading to reduced patient disability in the short-term and more clot removal treatments performed.

Stroke is a leading cause for serious long-term disability in the United States. The economic burden of stroke exceeds an astonishing $100 billion per annum (Girotra, Lekoubou et al., 2020). Stroke incidence and associated costs are rising drastically due to an aging population and increasing unhealthy lifestyle. NICO.LAB is dedicated to playing a crucial role in reducing stroke related costs and improving quality of life of patients by combining human and artificial intelligence to revolutionize emergency stroke care.

About

NICO.LAB was founded in 2015 as a spin-off from the Amsterdam University Medical Center in the Netherlands. An international team of researchers, developers and physicians connects human & artificial intelligence to empower physicians in emergency care with end-to-end solutions. The company is active in Europe, Asia, Australia and the United States. StrokeViewer consists of a set of tools to support the entire stroke workflow. StrokeViewer LVO is the first with FDA approval. Local availability of StrokeViewer functionality is subject to applicable CE marking, TGA and FDA approval (actual status on www.nico-lab.com).

References
Saver, 2006 – “Time is brain—quantified.” Stroke 37.1 (2006): 263-266
FDA K200873 – StrokeViewer LVO is approved as HALO under FDA submission number K200873
Girotra, Lekoubou et al. 2020 – “A contemporary and comprehensive analysis of the costs of stroke in the United States” JNS-journal, volume 410, 116643

Contact

Mike Moolenbel
Head of Marketing & Communications
mmoolenbel@nico-lab.com
+31642228955

Related Images

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NICO.LAB
NICO.LAB logo

strokeviewer.jpg
StrokeViewer
StrokeViewer logo

merel-boers-ceo-founder.jpg
Merel Boers (CEO & Founder)
Merel Boers (CEO & Founder)

strokeviewer-lvo.jpg
StrokeViewer LVO
StrokeViewer on smartphone

Related Links

NICO.LAB website

NICO.LAB LinkedIn page

Related Video

SOURCE NICO.LAB B.V.

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The CARTO® 3 System and THERMOCOOL SMARTTOUCH® SF Catheter allow for a patient-tailored ablation approach, resulting in high long-term effectiveness in persistent atrial fibrillation patients*

Date of Release: Oct. 6, 2020

IRVINE, Calif. /PRNewswire/ — Johnson & Johnson Medical Devices Companies^** today announced the United States Food and Drug Administration (FDA) approval of Biosense Webster, Inc.’s THERMOCOOL SMARTTOUCH^® SF Ablation Catheter for the treatment of persistent atrial fibrillation (persistent AF).^† The approval is based on results of a prospective, multi-center study (PRECEPT) which met primary safety and effectiveness endpoints and demonstrated 80 percent of persistent AF patients experienced clinical success at 15 months after ablation therapy using the THERMOCOOL SMARTTOUCH SF Catheter with the CARTO VISITAG™ Module.^‡1,2 In addition, patients experienced clinically meaningful improvement in quality of life and the study showed significant reduction in healthcare resource utilization post-ablation.^1,2

Atrial fibrillation (AF) is a significant public health issue affecting the health of millions of people and placing a critical burden on healthcare systems. Persistent AF is defined as continuous AF that lasts for more than seven days and up to one year. The management of persistent AF aims to prevent AF recurrence and associated disabilities while reducing side effects from treatment.

“Every patient and every arrhythmia are unique,” said Dr. Francis Marchlinski***, Director of Electrophysiology, University of Pennsylvania Health System. “This approval and the PRECEPT data provide evidence to support a tailored approach using the CARTO^® 3 System and THERMOCOOL SMARTTOUCH SF Catheter to treat persistent AF patients, who are more at risk for stroke and other complications from their AF.”

The PRECEPT study is the first prospective, multi-center investigational device exemption study designed to evaluate the safety and effectiveness of radiofrequency (RF) catheter ablation in patients with persistent AF, and was conducted using the THERMOCOOL SMARTTOUCH SF Catheter. Results of the PRECEPT study demonstrated 80 percent of persistent AF patients experienced clinical success at 15 months after ablation therapy and 86 percent experienced freedom from repeat procedures at 15 months.³ The CARTO 3 System and THERMOCOOL SMARTTOUCH SF Catheter allow for a patient-tailored ablation approach, resulting in high long-term effectiveness in a more advanced persistent AF patient group (continuous AF > 7 days < 1 year).^*2

Radiofrequency ablation with the THERMOCOOL SMARTTOUCH SF Catheter led to a clinically meaningful improvement in Quality of Life (QOL), as well as a reduction in antiarrhythmic drug (AAD) use, cardioversion and hospitalization in persistent AF patients.^§3

“Persistent AF patients face a higher risk of complications such as stroke, heart failure, and death,” said Uri Yaron, Worldwide President of Biosense Webster, Inc. “This approval and data from the PRECEPT study help to further our commitment to advancing AF treatment, providing electrophysiologists with state-of-the-art options for their patients.”

The PRECEPT study enrolled a total of 381 patients with documented symptomatic persistent AF who did not respond or were intolerant of one or more AADs (Class I or III). The study was conducted at 27 sites across the United States and Canada.¹ The primary effectiveness endpoint was freedom from documented recurrence of atrial flutter/atrial tachycardia episodes of 30 seconds or longer and freedom from additional five failure modes: acute procedural failure, use of a non-study catheter, repeat procedures, use of new/higher dose antiarrhythmic drugs, surgical AF ablation.¹ A tailored ablation strategy was used, allowing for pulmonary vein isolation (PVI) and additional left atrial ablations (PVI+) at the operator’s discretion based on the patient’s disease state.¹ The study resulted in a 4.7 percent primary adverse event (PAE) rate which is comparable to PAE rates reported in paroxysmal AF studies using CF-sensing RF catheters.^4,5,6

About Atrial Fibrillation

Atrial fibrillation (AF) is the most common type of cardiac arrhythmia (abnormal heart rhythm) and affects nearly one percent of the population.⁷ During AF, the upper chambers of the heart, the atria, beat rapidly or in an uncontrolled manner, which can feel like a flutter. When the heart beats erratically, it does not pump blood as efficiently as it should. When oxygen is not being properly delivered to all parts of the body, the patient may feel ill or experience other AF symptoms. AF may not be life-threatening; however, it is important to seek treatment to control the symptoms, as AF can lead to stroke.

As with any medical treatment, individual results may vary. Only a cardiologist or electrophysiologist can determine whether ablation is an appropriate course of treatment. There are potential risks including bleeding, swelling or bruising at the catheter insertion site, and infection. More serious complications are rare, which can include damage to the heart or blood vessels; blood clots (which may lead to stroke); heart attack; or death. These risks need to be discussed with your doctor and recovery takes time. The success of this procedure depends on many factors, including your physical condition and your body’s ability to tolerate the procedure. Use care in the selection of your doctors and hospital, based on their skill and experience.

About Biosense Webster, Inc.

Biosense Webster, Inc. is the global market leader in the science and technology behind the diagnosis and treatment of cardiac arrhythmias. Part of the Johnson & Johnson Family of Companies, the specialized medical-technology company is headquartered in Irvine, Calif., and works across the world to advance the tools and solutions that help electrophysiologists identify, treat, and deliver care. Learn more at www.biosensewebster.com and connect on LinkedIn and Twitter.

About Johnson & Johnson Medical Devices Companies

As the world’s most comprehensive medical devices business, we are building on a century of experience, merging science and technology, to shape the future of health and benefit even more people around the world. With our unparalleled breadth, depth and reach across surgery, orthopedics, vision and interventional solutions, we’re working to profoundly change the way care is delivered. We are in this for life. For more information, visit www.jnjmedicaldevices.com.

Cautions Concerning Forward-Looking Statements

This press release contains “forward-looking statements” as defined in the Private Securities Litigation Reform Act of 1995 regarding THERMOCOOL SMARTTOUCH^® SF Ablation Catheter. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Biosense Webster, Inc., any of the other Johnson & Johnson Medical Devices Companies and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: uncertainty of commercial success; challenges to patents; competition, including technological advances, new products and patents attained by competitors; changes to applicable laws and regulations, including global health care reforms; changes in behavior and spending patterns of purchasers of health care products and services; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson’s Annual Report on Form 10-K for the fiscal year ended December 29, 2019, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in the company’s most recently filed Quarterly Report on Form 10-Q, and the company’s subsequent filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. Neither Biosense Webster, Inc., the Johnson & Johnson Medical Devices Companies, nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.

*In a prospective, multicenter study (PRECEPT, n=333) protocol defined primary effectiveness was the freedom from documented AF/AT/AFL recurrence ≥30 s.
^† The THERMOCOOL SMARTOUCH® SF Catheter is indicated for drug refractory recurrent symptomatic persistent atrial fibrillation (AF) (continuous AF > 7 days but < 1 year), refractory or intolerant to at least 1 Class I or III AAD, when used with the CARTO® 3 System.
^‡ In a prospective, multicenter study (PRECEPT, n=333) where clinical success is defined as freedom from documented symptomatic AF/AT/AFL recurrence.
^§ In a prospective, multicenter study (PRECEPT, n=333), improvements based on mean AFEQT composite and subscores seen from 6– 15 months, exceeding Clinically Important Difference (±5 points). Class I/III AAD use was reduced from 97% to 25%, incidence of cardioversion decreased from 62% to 10%, and the 15–month Kaplan–Meier estimate of freedom from hospitalization was 84%.
**Comprising the surgery, orthopedics, vision and interventional solutions businesses within Johnson & Johnson’s Medical Devices segment
***Dr. Marchliniski is a paid consultant to Biosense Webster, Inc.

¹ Mansour M, Calkins H, Osorio J, et al. Persistent atrial fibrillation ablation with a contact force sensing catheter: the prospective multicenter PRECEPT trial. Presented at: Heart Rhythm Society Scientific Session Virtual Meeting; May 2020.
² Mansour M, Calkins H, Osorio J, et al. Persistent Atrial Fibrillation Ablation with Contact Force–Sensing Catheter. J Am Coll Cardiol EP. 2020 Aug, 6 (8) 958-969.
³ Natale A, Calkins H, Osorio J, et al. (2020). Positive Clinical Benefit on Patient Care, Quality of Life and Symptoms After Radiofrequency Ablation with Contact Force in Persistent Atrial Fibrillation: Analyses from PRECEPT. Poster presentation at the European Society of Cardiology Scientific Session, August 29 – September 2, 2020.
⁴ PRECEPT IDE G140102.
⁵ Natale A, Reddy VY, Monir G, et al. Paroxysmal AF catheter ablation with a contact force sensing catheter: results of the prospective, multicenter SMART-AF trial. J Am Coll Cardiol. 2014;64(7):647-656.
⁶ Chinitz LA, Melby DP, Marchlinski FE, et al. Safety and efficiency of porous-tip contact-force catheter for drug-refractory symptomatic paroxysmal atrial fibrillation ablation: results from the SMART SF trial. Europace. 2018;20(FI_3):f392-f400.
⁷ Johan E.P. Waktare, MB, ChB, MRCP, Atrial Fibrillation, AHA Journals.org.

© Biosense Webster, Inc. 2020   151997-200904

SOURCE Biosense Webster, Inc.

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Date of Release: Sept. 11, 2020

TAIPEI and SAN DIEGO /PRNewswire/ — Senhwa Biosciences, Inc. (TPEx: 6492), a clinical-stage biopharmaceutical company focused on next generation DNA Damage Response (DDR) therapeutics for the treatment of cancer, announced today that the first patient with severe COVID-19 demonstrated remarkable recovery after treatment with the Company’s investigational drug, Silmitasertib.

On August 27, 2020, the Food and Drug Administration (FDA) approved the first emergency IND and authorized use of Silmitasertib in a patient with severe COVID-19 pneumonia requiring supplemental oxygen. The patient had been treated with multiple therapeutics, including Remdesivir, Dexamethasone, Ceftriaxone, Azithromycin and Enoxaparin within two weeks, but remained hypoxic and required up to 2 liters of supplemental oxygen daily. As none of the available therapeutics worked well for this patient, the investigator decided to try Senhwa’s investigational drug, Silmitasertib.  Within 24 hours of the first dose the patient showed significant clinical improvement and the oxygen requirement was weaned to room air. The patient was discharged from the hospital five days after starting Silmitasertib.

Marilyn Glassberg Csete, MD, Chief of Pulmonary, Critical Care, and Sleep Medicine at University of Arizona College of Medicine/Banner – University Medical Center Phoenix and Esa Rayyan, DO, her co-investigator, are now looking for five to ten more patients with severe COVID-19 to treat with Silmitasertib with a plan for a randomized clinical trial in the near future.

“This is the first person in the world to receive Silmitasertib for this novel coronavirus, and it seems to have worked,” said Dr. John Soong, the Chief Medical Officer of Senhwa Biosciences. “It is only one case, and it is still early to know how well the treatment will do in others, but if the same result is repeated in other patients, it will give us an opportunity to significantly reduce the average time of COVID-19 patient hospitalization and reduce the burden on healthcare systems,” Soong added.

“This first patient that received Silmitasertib was discharged in five days! We are encouraged by the patient’s strong response to Silmitasertib and will make every effort to provide our drug to critically ill COVID-19 patients,” said Benny T. Hu, Chairman of Senhwa Biosciences.

Banner – University Medical Center Phoenix is also planning to start a Phase 2, Investigator-Initiated Trial (IIT) of 40 patients. Another Phase 2 IIT will be conducted at the Center for Advanced Research and Education (CARE) in Gainesville, Georgia. The CARE trial will seek to enroll 10 patients once it is approved by the FDA.

About Silmitasertib
Silmitasertib is a first-in-class small molecule drug that targets CK2 and acts as a CK2-inhibitor. Silmitasertib is safe and well-tolerated in humans.  To date, three Phase I trials of Silmitasertib in cancer patients have been completed; currently, there are one ongoing Phase I and two ongoing Phase II studies of Silmitasertib.  In December 2016, Silmitasertib was granted Orphan Drug Designation by the US FDA for the treatment of Cholangiocarcinoma. In July 2020, Silmitasertib was granted Rare Pediatric Disease Designation (RPD) in Medulloblastoma by the US FDA. An eIND was granted by the US FDA on August 27, 2020 to Dr. Rayyan for use in the COVID patient treated at BUMCP.

About Senhwa Biosciences, Inc.
Senhwa Biosciences, Inc. is a leading clinical stage company focusing on developing first-in-class, next generation DDR therapeutics for patients with unmet medical needs in oncology.  Headquartered in Taiwan, with an operational base in San Diego, California, Senhwa is well positioned to oversee the development of their compounds.

Development is currently focused on two lead products Silmitasertib (CX-4945) and Pidnarulex (CX-5461) with novel mechanisms of action and for multiple indications.  Clinical trials are ongoing in Australia, Canada, United States, Korea, and Taiwan, with more currently in development. 

Visit Senhwa Biosciences for more details: www.senhwabio.com

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Fully transparent, anti-fog masks available to help healthcare providers and essential workers to protect themselves, communicate better, and ease confusion

“We are ready to join the fight against the current pandemic.” — ALLYSA DITTMAR, PRESIDENT OF CLEARMASK

DATE OF RELEASE: Aug, 18, 2020

BALTIMORE/PRNewswire/ — ClearMask LLC, a privately held American medical supply company, announced today that it received the U.S. Food and Drug Administration’s 510(k) clearance on April 6, 2020, for its ClearMask™, a fully transparent surgical mask.

ClearMask™ is the world’s first FDA-cleared, fully transparent surgical mask that can be used in hospitals, clinics, schools, retail, hospitality, and other settings. The mask is optimized for maximum clarity and comfort, and meets applicable ASTM Level 3 requirements for fluid resistance and flammability, which offers a high level of protection for medical use in environments such as operating rooms. 

In this disheartening time as the world fights against the COVID-19 pandemic, the ClearMask™ helps provide protection while bringing much-needed relief through a reassuring smile and familiar face among fear, confusion, and suffering. In addition to blocking particles or droplets with its fully transparent, anti-fog plastic barrier, the ClearMask™ helps improve visual communication, which may help avoid costly errors and adverse outcomes. Transparent communication during the customer experience can be critical in establishing rapport and earning trust, while assuring safety as a priority. 

The company, consisting of Johns Hopkins University graduate students and alumni, started developing the transparent mask in 2017 after their deaf co-founder experienced an adverse experience during her surgery. Traditional surgical masks blocked her providers’ faces, impeding effective communication and safety. 

“After three years of research, development, and testing, we are thrilled to bring a human-centered mask to everyone who needs it, especially those who can benefit from improved visual communication, such as children, older adults, deaf and hard of hearing people, and those who do not speak the same language. The ClearMask™ is well-positioned to join the fight against the current pandemic,” said Allysa Dittmar, President of ClearMask. 

The ClearMask™ is the first product in ClearMask LLC’s growing portfolio of novel masks to receive FDA clearance as a Class II medical device. To fulfill the current and rising demand as the company ramps up production of the FDA-cleared mask, the company is currently offering a non-medical, consumer face mask that similarly helps to improve visual communication and provide protection at a lower price. The consumer masks have provided much relief to different communities in need, including state emergency management agencies and essential workers. 

To date, the company has provided the masks in bulk volumes, typically in the tens to hundreds of thousands. The consumer masks can now be purchased through ClearMask’s website at buy.theclearmask.com, starting at a box of 24 masks. The company has partnered with several distributors, including Cardinal Health Canada, McKesson, Oaktree Products, and Grainger to help get the masks out to as many people as possible. 

“Regulatory clearance and mass production are two significant milestones in ClearMask’s mission to get the ClearMask out to as many people as possible,” said Aaron Hsu, CEO of ClearMask. “This achievement is a testament to our company’s hard work and commitment to serving different communities in need during this time.”

About ClearMask ClearMask, LLC is an American-based medical supply company that focuses on improving communication while making connections more human. Its products may help reduce errors and increase satisfaction by facilitating visual communication for all. For more information, please visit www.theclearmask.com.

Media Contact:
Margaret Justus
281-250-8253
press@theclearmask.com

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SOURCE ClearMask LLC

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Texas Cardiac Arrhythmia Institute at St. David’s Medical Center, Los Robles Health System perform near simultaneous implantation of WATCHMAN FLX™ Left Atrial Appendage Closure (LAAC)

DATE OF RELEASE: Aug. 7, 2020

AUSTIN, Texas /PRNewswire/ — On Aug. 5, 2020, physicians at the Texas Cardiac Arrhythmia Institute (TCAI) at St. David’s Medical Center in Austin, Texas, and Los Robles Health System in Thousand Oaks, California, became the first in the nation to implant the only FDA-approved device for the reduction of stroke risk in patients with non-valvular atrial fibrillation (A Fib). The physicians at these hospitals, both HCA Healthcare facilities, performed near simultaneous implantation of the WATCHMAN FLX^TM Left Atrial Appendage Closure (LAAC).

Andrea Natale, M.D., F.H.R.S., F.A.C.C., F.E.S.C., cardiac electrophysiologist and executive medical director of TCAI, and Saibal Kar, M.D., interventional cardiologist at Los Robles Health System, performed the first procedure in Thousand Oaks, California, while Rodney Horton, M.D., cardiac electrophysiologist at TCAI, performed the first case in Austin, Texas. Additionally, Dr. Kar was the principal investigator for the preclinical PINNACLE FLX trial prior to FDA approval, which evaluated the performance of the WATCHMAN FLX device as an alternative to long-term, non-vitamin K antagonist oral anticoagulants (NOACs) and other oral anticoagulant medications.

“The new device is built upon the most studied and implanted LAAC device in the world as a treatment option for people with A Fib not caused by a heart valve problem, also known as non-valvular A Fib,” Dr. Kar said. “It allows us to help treat more patients safely and effectively to ensure the best outcomes.”

Up to six million Americans are estimated to be affected by A Fib, an irregular heartbeat that feels like a quivering heart. People with A Fib have a five times greater risk of stroke than those with normal heart rhythms.

“This device serves as a safe and effective alternative to reduce the risk of stroke for patients with non-valvular A Fib, especially those with a compelling reason not to be on blood thinners,” Dr. Horton said.

This technology is designed to reduce the risk of stroke by permanently closing off an area of the heart called the left atrial appendage (LAA) to keep harmful blood clots that can form in the LAA from entering the bloodstream and potentially causing a stroke. By closing off the LAA, the risk of stroke may be reduced and, over time, patients may be able to stop taking blood thinners, such as warfarin.

“The rounded design allows us to safely enter, and maneuver within, the LAA, resulting in optimal placement and long-term stability,” Dr. Natale said. “It is also available in broader size options than the previous generation device, which allows us to treat a wider range of patients.”

The procedure is done under general anesthesia and takes about an hour. Patients commonly stay in the hospital overnight and leave the next day.

Media Contacts:
Erin Ochoa
St. David’s HealthCare
EOchoa@EChristianPR.com
512-788-1616

Daphne Yousem
Los Robles Health System
805-390-7111
Daphne.Yousem@HCAHealthcare.com

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– Evrysdi represents the first at home, oral treatment approved for infants, children and adults with all SMA types-

– Marketing Authorization Application submission to European Medicines Agency is imminent –

– Risdiplam is the first approved therapy discovered and developed from PTC Therapeutics’ proprietary splicing platform –

SOUTH PLAINFIELD, N.J., Aug. 7, 2020 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has approved Evrysdi™ (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older. Evrysdi showed clinically meaningful improvements in motor function and obtainment of developmental milestones across two trials in patients age two months and above and across all levels of disease severity, including types 1, 2, and 3 SMA. Infants achieved key motor milestones not normally seen in the natural course of the disease, such as the ability to sit without support. Evrysdi also preserved vital functions and improved survival at 12 months. In particular, Evrysdi improved survival without permanent ventilation compared to natural history when measured at 12 and 23 months.

“Today marks an incredibly important moment for the broader SMA patient community that had been in dire need of safe and effective treatment options,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. “We are appreciative of the patients, physicians and others involved with its development, and our partners at the SMA Foundation and Roche. We are proud that the first oral treatment for spinal muscular atrophy patients was discovered through our splicing platform. I believe that this is one of the most innovative products in the pharmaceutical industry and we are gratified to have played a key role in its discovery and development.”

Evrysdi has been studied in a robust clinical trial program in SMA with more than 450 patients and subjects. The program includes infants aged 2 months to adults aged 60 with a range of symptoms and motor function including patients with scoliosis or joint contractures. Patients previously treated for SMA with other medications were also included. The approval is based on results from two clinical studies designed to represent a broad spectrum of patients living with SMA: FIREFISH in symptomatic infants aged 2 to 7 months; and SUNFISH, in children and adults aged 2 to 25 years. SUNFISH is the first and only placebo-controlled trial to include adults with types 2 and 3 SMA. 

“We started looking for treatments for SMA more than a decade ago,” said Loren Eng, President of the SMA Foundation. “PTC was one of the companies that embraced and remained committed to the idea of developing a truly innovative therapeutic for this disease. We are proud to be part of the collaboration that brought the first oral SMA treatment for patients.”

In FIREFISH, 41% (7/17) of infants treated with the therapeutic dose achieved the ability to sit without support for at least 5 seconds as measured by the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Additionally, 90% (19/21) of infants were alive without permanent ventilation at 12 months of treatment and reached 15 months of age or older. As described in the natural history of untreated infantile-onset SMA, infants would not be expected to be able to sit independently and only 25 percent of infants would be expected to survive without permanent ventilation beyond 14 months of age. In SUNFISH, children and adults treated with Evrysdi experienced a statistically significant and clinically meaningful improvement in motor function at 12 months (1.55 point mean difference; p = 0.0156) compared to placebo, as measured by a change in baseline on the Motor Function Measure-32 (MFM-32) scale.

Evrysdi demonstrated a favorable efficacy and safety profile, with the safety profile established across the FIREFISH and SUNFISH trials. The most common adverse reactions were fever, diarrhea, and rash in later-onset SMA. In infantile-onset SMA, the most common adverse events were similar and also included upper respiratory tract infection, pneumonia, constipation, and vomiting. There were no treatment-related safety findings leading to withdrawal from any study. Ophthalmological monitoring is not required.

Evrysdi will be available in the United States within two weeks for direct delivery to patients’ homes through Accredo Health Group Inc., an Express Scripts specialty pharmacy.

Evrysdi will be marketed in the United States by Genentech, a member of the Roche Group, and received an orphan drug designation from FDA in 2017. Outside the United States, Roche holds global commercialization rights to risdiplam, and the European Medicines Agency (EMA) previously granted PRIME (Priority Medicines) designation to risdiplam in 2018 for the treatment of people with SMA and an orphan drug designation in 2019. The submission of a marketing authorization application (MAA) to EMA for risdiplam is pending. At this time, Roche has submitted applications for approval in Brazil, Chile, China, Indonesia, Russia, South Korea and Taiwan.

Evrysdi is a product of the SMA collaboration between PTC, the SMA Foundation, and Roche.

About Evrysdi™ (risdiplam)

Evrysdi is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi is designed to distribute evenly to all parts of the body, including the central nervous system (CNS). Evrysdi is administered daily at home in liquid form by mouth or feeding tube.

About the Clinical Studies

FIREFISH (NCT02913482)

FIREFISH, an open-label, two-part pivotal study, was designed to assess Evrysdi safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) in patients aged 1 to 7 months with Type 1 SMA. Part 1 evaluated several doses of Evrysdi and determined the therapeutic dose of 0.2 mg/kg for Part 2. In Part 1, after 12 months of Evrysdi treatment:

• 41% (7/17) of infants treated with the therapeutic dose achieved the ability to sit without support for at least 5 seconds as measured by the BSID-III gross motor scale.
• 90% (19/21) of all infants were alive without permanent ventilation* and reached 15 months of age or older.
• 81% (17/21) of all patients were alive without permanent ventilation* after a minimum of 23 months of treatment and reached an age of 28 months or older (median 32 months; range 28-45 months)

*Permanent ventilation defined as tracheostomy or ≥16 hours of non-invasive ventilation per day or intubation for ≥21 consecutive days in the absence of, or following the resolution of, an acute reversible event.

SUNFISH (NCT02908685)

SUNFISH, a two-part, placebo-controlled, multicenter pivotal trial, was designed to assess Evrysdi safety, tolerability, efficacy, PK and PD in people with type 2 or 3 SMA aged 2 to 25, including those with scoliosis (67% in Part 2) and joint contractures at baseline. In Part 2, after 12 months, Evrysdi treatment led to:

• A clinically meaningful and statistically significant improvement in motor function among children and adults, as measured by a change in baseline in the MFM-32 total score (1.55 point mean difference; p = 0.0156), at 12 months as compared to placebo (1.36 points [risdiplam treatment arm -95% CI: 0.61, 2.11]; [placebo treatment arm: -0.19 points 95% CI: 1.22, 0.84]). MFM-32 assesses 32 different motor functions across a wide range of people with SMA.
• Improved upper limb motor function compared to baseline, as measured by the Revised Upper Limb Module (RULM), a secondary independent motor function endpoint of the study (1.59 point difference; p=0.0028).

Clinical Trial Safety Data

The safety profile of Evrysdi was established across FIREFISH and SUNFISH pivotal trials. The most common adverse reactions in later-onset SMA (incidence of at least 10% of patients treated with Evrysdi and more frequently than control) were fever, diarrhea, and rash. The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, the most common adverse reactions (incidence of at least 10%) were upper respiratory tract infection, pneumonia, constipation, and vomiting.

About the Evrysdi Clinical Trial Program

In addition to FIREFISH and SUNFISH, Evrysdi is being evaluated in a broad range of people with SMA, including in:

• JEWELFISH (NCT03032172): an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi. Recruitment for this study is complete with 174 people enrolled.
• RAINBOWFISH (NCT03779334): an open-label, single-arm, multicenter study investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of Evrysdi in infants (~n=25), from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study is currently recruiting.

About Spinal Muscular Atrophy (SMA)

Spinal muscular atrophy (SMA) is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting approximately one in 11,000 babies¹. SMA leads to the progressive loss of nerve cells in the spinal cord that control muscle movement. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

SMA is caused by a mutation in the survival motor neuron 1 (SMN1) gene that results in a deficiency of SMN protein. SMN protein is found throughout the body and increasing evidence suggests SMA is a multi-system disorder and the loss of SMN protein may affect many tissues and cells, which can stop the body from functioning.

About PTC Therapeutics, Inc.

PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.

For More Information:
Investors:
Alex Kane
+1 (908) 912-9643
akane@ptcbio.com

Media: 
Jane Baj
+1 (908) 912-9167 
jbaj@ptcbio.com 

Forward-Looking Statements:

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historical fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC; the timing of commercial availability and potential methods of distribution for Evrysdi; advancement of PTC’s joint collaboration program in SMA, including any potential regulatory submissions, regulatory approvals or commercial prospects;  PTC’s strategy, future operations, future financial position, future revenues and, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words “guidance”, “plan,” “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions.

PTC’s actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC’s products or product candidates that PTC commercializes or may commercialize in the future, including Evrysdi; the enrollment, conduct, and results of studies under the SMA collaboration and events during, or as a result of, the studies that could delay or prevent further development under the program, including any potential regulatory submissions and potential commercialization with regards to Evrysdi; the eligible patient base and commercial potential of Evrysdi or any of PTC’s other product candidates; and the factors discussed in the “Risk Factors” section of PTC’s most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC’s other filings with the SEC. You are urged to carefully consider all such factors.

As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product or product candidate will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Evrysdi.

The forward-looking statements contained herein represent PTC’s views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

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