GlobeNewswire by notified

FDA approves Roche’s Evrysdi for use in babies under two months with spinal muscular atrophy (SMA)

Share
  • Approval based on interim RAINBOWFISH data which show pre-symptomatic babies treated with Evrysdi for at least one year were able to sit, stand and walk
  • Prescribing information updated with FIREFISH data showing the majority of symptomatic babies treated with Evrysdi for at least two years could sit for at least five seconds
  • Evrysdi has proven efficacy in babies, children and adults with more than 5,000 patients treated to date

Basel, 31 May 2022 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to include babies under two months old with spinal muscular atrophy (SMA). The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting and standing with half walking after 12 months of treatment. Evrysdi is now approved in the US to treat SMA in children and adults of all ages.

Of the babies with 2 or 3 copies of the SMN2 gene (n=6), 100% were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% of infants could walk independently. All infants were alive at 12 months without permanent ventilation.

“The approval of Evrysdi for pre-symptomatic babies is particularly important, as early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones,” said Richard Finkel, M.D., RAINBOWFISH Principal Investigator and Director of the Experimental Neuroscience Program at St. Jude Children’s Research Hospital. “With the inclusion of SMA in newborn screening programmes, this approval provides the opportunity to start treating at home with Evrysdi soon after the diagnosis is confirmed.”

As part of the label extension, the Evrysdi prescribing information has also been updated to include recent two-year pooled data from Parts 1 and 2 of the FIREFISH study, which demonstrate long-term efficacy and safety in symptomatic infants with Type 1 SMA. The study enrolled babies aged 1-7 months and after two years of treatment with Evrysdi at the recommended dose (n=58), 60% of infants were able to sit without support* for five seconds, 40% for 30 seconds and 28% of infants were able to stand.**

Without treatment, infants do not achieve these milestones in the natural history of the disease. There were no treatment-related adverse events leading to withdrawal. The most common adverse reactions were upper respiratory tract infection (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting, and cough.

“The priority review and subsequent approval of Evrysdi for babies under two months of age speaks to the urgent ongoing need for additional treatment options for babies with SMA,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA from pre-symptomatic newborns to older adults. We are proud of this achievement, which has the potential to make a real difference to those living with SMA and their caregivers.”

Evrysdi is approved in 81 countries and the dossier is under review in a further 27 countries. More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

About Evrysdi® (risdiplam)
Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube.

Evrysdi is designed to treat SMA by increasing and sustaining the production of the survival motor neuron (SMN) protein in the central nervous system (CNS) and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.

Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S Food and Drug Administration in 2017. In 2021, Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research award for Drug Discovery. Evrysdi is currently approved in 81 countries and the dossier is under review in a further 27 countries.


Evrysdi is currently being evaluated in five multicentre trials in people with SMA:

  • FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of risdiplam in infants and determining the dose for Part 2. Part 2 is a pivotal, single-arm study of risdiplam in 41 infants with Type 1 SMA treated for 2 years, followed by an open-label extension. Enrolment for Part 2 was completed in November 2018. The primary objective of Part 2 was to assess efficacy as measured by the proportion of infants sitting without support after 12 months of treatment, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III) (defined as sitting without support for 5 seconds). The study met its primary endpoint.
  • SUNFISH (NCT02908685) – SUNFISH is a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using the total score of Motor Function Measure 32 (MFM-32) at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. The study met its primary endpoint.
  • JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics in people with SMA aged 6 months to 60 years who previously received other investigational or approved SMA therapies prior to receiving Evrysdi. The study has completed recruitment (n=174).
  • RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicentre study, investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of risdiplam in babies (~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 fully enrolled.
  • MANATEE (NCT05115110) – a global phase 2/3 clinical study to evaluate the safety and efficacy of GYM329 (RO7204239), an anti-myostatin molecule targeting muscle growth, in combination with risdiplam for the treatment of SMA in patients 2-10 years of age. The FDA Office of Orphan Products Development granted GYM329 Orphan Drug Designation for the treatment of patients with SMA in December 2021. The study is commencing recruitment in Q1 2022.

About SMA
SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. 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.

About Roche in Neuroscience
Neuroscience is a major focus of research and development at Roche. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases.

Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today.

About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.

In recognizing our endeavor to pursue a long-term perspective in all we do, Roche has been named one of the most sustainable companies in the pharmaceuticals industry by the Dow Jones Sustainability Indices for the thirteenth consecutive year. This distinction also reflects our efforts to improve access to healthcare together with local partners in every country we work.

Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan.

For more information, please visit www.roche.com.

All trademarks used or mentioned in this release are protected by law.

References
* Bayley Scales of Infant and Toddler Development – Third Edition
** Hammersmith Infant Neurological Examination (HINE-2)

Roche Group Media Relations
Phone: +41 61 688 8888 / e-mail: media.relations@roche.com

Hans Trees, PhD
Phone: +41 61 687 41 47

KarstenKleine
Phone: +41 61 682 28 31
Nina Mählitz
Phone: +41 79 327 54 74

Nathalie Meetz
Phone: +41 79 771 05 25
Dr. Barbara von Schnurbein
Phone: +41 61 687 89 67
SileiaUrech
Phone: +41 79 935 81 48

Roche Investor Relations

Dr. Bruno Eschli
Phone: +41 61 68-75284
e-mail: bruno.eschli@roche.com

Dr. Sabine Borngräber
Phone: +41 61 68-88027
e-mail: sabine.borngraeber@roche.com
Dr. Birgit Masjost
Phone: +41 61 68-84814
e-mail: birgit.masjost@roche.com
Dr. Gerard Tobin
Phone: +41 61 68-72942
e-mail: gerard.tobin@roche.com

Investor Relations North America

Loren Kalm
Phone: +1 650 225 3217
e-mail: kalm.loren@gene.com

Attachment

To view this piece of content from www.globenewswire.com, please give your consent at the top of this page.
To view this piece of content from ml-eu.globenewswire.com, please give your consent at the top of this page.

About GlobeNewswire by notified

GlobeNewswire by notified
GlobeNewswire by notified
One Liberty Plaza - 165 Broadway
NY 10006 New York

https://notified.com

GlobeNewswire by notified is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.

Subscribe to releases from GlobeNewswire by notified

Subscribe to all the latest releases from GlobeNewswire by notified by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from GlobeNewswire by notified

Millicom increases Tigo Panama ownership to 100%29.6.2022 23:36:16 CEST | Press release

Millicom increases Tigo Panamaownership to 100% Luxembourg, June 29, 2022 – Millicom closed today the previously-announced transaction to acquire the remaining 20% interest in Telecomunicaciones Digitales, S.A. (formerly known as Cable Onda S.A. or “Tigo Panama”) reflecting an exercise of the founders’ liquidity option as provided for in the Shareholders’ Agreement, which was entered into in 2018. As a result, Millicom now owns 100% of Tigo Panama, the leader in Panama’s telecommunications market. For further information, please contact: Press: Yocasta Valdez, Sr. Manager Digital Media & Communications press@millicom.com Investors: Michel Morin, VP Investor Relations Sarah Inmon, Director Investor Relations investors@millicom.com About Millicom Millicom (NASDAQ U.S.: TIGO, Nasdaq Stockholm: TIGO_SDB) is a leading provider of fixed and mobile telecommunications services in Latin America. Through our TIGO® and Tigo Business® brands, we provide a wide range of digital services and product

Kalera Public Limited Company announces its share structure following the business combination with Agrico Acquisition Corp.29.6.2022 16:30:19 CEST | Press release

ORLANDO, Fla., June 29, 2022 (GLOBE NEWSWIRE) -- Kalera Public Limited Company ( “Kalera”) and Agrico Acquisition Corp. (“Agrico”) today announced the completion of their previously announced business combination. The combined company will retain the Kalera name and will commence trading ordinary shares (“Kalera Shares”) and warrants (“Kalera Warrants”) on Nasdaq under the new ticker symbols “KAL” and “KALWW”, respectively, on Wednesday, June 29, 2022. The business combination was approved at a special meeting of the shareholders of Kalera S.A. on June 28, 2022. Update on Share Structure Pursuant to the Sponsor Support Agreement entered into on January 30, 2022, upon the consummation of the business combination approximately 1.8 million shares (50%) of Agrico Class B common stock were first converted into Agrico Class A common stock on a one-for-one basis, then into Kalera Shares on a one-for-one basis. The remaining 50% of the Agrico Class B common stock was forfeited to Agrico. In co

Business School Graduates Enter White-Hot Job Market as Employers Signal Growth, Confidence in Their Credentials29.6.2022 16:00:00 CEST | Press release

MBA and business master’s degrees remain hot commodity despite recession fears and COVID challenges RESTON, Va., June 29, 2022 (GLOBE NEWSWIRE) -- The Graduate Management Admission Council (GMAC), a global association of leading graduate business schools, today released its annual hiring report, the GMAC Corporate Recruiters Survey – 2022 Summary Report. The report explores the state of employer demand for graduate business school talent (MBA and business master’s degree recipients) in the context of the COVID-19 pandemic and includes responses from nearly 1,000 corporate recruiters and staffing firms around the world. While rising inflation and the war in Ukraine were just at the onset during the time when the survey was conducted in February and March 2022, hiring projections of graduate management education (GME) graduates remain bullish this year, with 92 percent of corporate recruiters expecting to hire newly minted MBAs. Promisingly, 2 in 3 responding corporate recruiters describ

AGC Biologics Named Manufacturing Company of the year by American Business Awards29.6.2022 15:09:00 CEST | Press release

Global CDMO honored for Growth in Mammalian, Microbial and Cell and Gene Manufacturing capabilities at Seattle, Boulder and Longmont sites SEATTLE, June 29, 2022 (GLOBE NEWSWIRE) -- AGC Biologics, a leading global Biopharmaceutical Contract Development and Manufacturing Organization (CDMO), today announced it was named Company of the Year in the “Large - Manufacturing” category in the 20th Annual American Business Awards®. The American Business Awards are the U.S.A.’s premier business awards program. Nicknamed the Stevies for the Greek word meaning “crowned,” the awards were presented to winners at a gala ceremony at the Marriott Marquis Hotel in New York on Monday, June 13. More than 230 professionals worldwide participated in the judging process to select this year’s American Business Award winners. AGC Biologics has a global pharmaceutical manufacturing network with seven sites across three continents. Over the last two years, the company has expanded its capabilities and reach with

PGS ASA: Approval and publication of prospectus – Listing of shares and Subsequent Offering29.6.2022 15:01:52 CEST | Press release

29 June, 2022 NOT FOR RELEASE, PUBLICATION OR DISTRIBUTION, IN WHOLE OR IN PART, DIRECTLY OR INDIRECTLY, IN OR INTO AUSTRALIA, CANADA, JAPAN, HONG KONG, SOUTH AFRICA OR THE UNITED STATES, OR ANY OTHER JURISDICTION IN WHICH THE RELEASE, PUBLICATION OR DISTRIBUTION WOULD BE UNLAWFUL. THIS ANNOUNCEMENT DOES NOT CONSTITUTE AN OFFER OF ANY OF THE SECURITIES DESCRIBED HEREIN. Reference is made to the previous stock exchange announcements by PGS ASA (“PGS” or the “Company”) regarding a successfully placed private placement (the "Private Placement") of new shares in the Company, raising gross proceeds of approximately NOK 800 million and a potential subsequent offering (the “Subsequent Offering) of up to 38,155,803 new shares at a subscription price of NOK 3.70, and the Company’s extraordinary general meeting’s approval of the same on 27 May 2022. The Private Placement consisted of one tranche of 74,200,000 new shares ("Tranche 1") and a second tranche of 142,016,216 new shares ("Tranche2"). T