Roche’s Evrysdi approved by European Commission as first and only at home treatment for spinal muscular atrophy
- Evrysdi has proven efficacy in adults, children and babies two months and older, as shown in two pivotal clinical trials
- Roche is actively engaging with health authorities in the EU to achieve broad and rapid access for people with SMA
- More than 3,000 patients now treated with Evrysdi in clinical trial, compassionate use and real-world settings
Basel, 30 March 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Commission (EC) has approved Evrysdi™ (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients two months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. SMA is a leading genetic cause of death in infants and 5q SMA is the most common form of the disease. The condition causes muscle weakness and progressive loss of movement and significant unmet need remains, particularly in adults living with this condition.
“Today’s approval of Evrysdi, the first and only SMA treatment with proven efficacy that can be taken at home, potentially transforms treatment options for a broad range of people with SMA living in the EU,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “By avoiding the need for in-hospital administration, Evrysdi can reduce the treatment burden on those living with SMA, their caregivers and healthcare systems. We thank the SMA community for their partnership, the trust they have placed in us and their unyielding commitment to achieve this significant milestone.”
The approval is based on data from two clinical studies, designed to represent a broad spectrum of people living with SMA: FIREFISH in symptomatic Type 1 infants aged 2 to 7 months and SUNFISH in symptomatic Type 2 and 3 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. Evrysdi demonstrated a favourable efficacy and safety profile, with the safety profile established across both trials.
"We welcome today’s approval of Evrysdi for people with SMA in Europe, and are proud of the role we have played in its development and of our partnership with Roche,” said Dr Nicole Gusset, President of SMA Europe. “A recent survey conducted by SMA Europe showed that a large proportion of people with SMA in the EU were not receiving an approved treatment which leaves them feeling helpless and frustrated. It is vital that we work together with health authorities, regulators and industry to ensure we can get this medicine to the patients who need it as soon as possible.’’
Roche is working closely with reimbursement and assessment bodies in European countries to enable broad and rapid access to patients in need. Evrysdi will be accessible to patients in Germany in the coming days and in France from early April through the cohort Temporary Authorization for Use. Reimbursement dossiers have been submitted in many countries in anticipation of today’s decision by the European Commission to minimise any delay in patient access.
The decision from the European Commission follows a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) in February 2021. The review was completed under the accelerated assessment pathway for medicines, which is offered to medicines deemed to be of major interest for public health and therapeutic innovation. This approval is applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation in 2019. Maintenance of Orphan Drug Designation was recently confirmed by the Committee for Orphan Medicinal Products based on the assumption of Evrysdi’s significant benefit over existing treatments. Evrysdi has been approved in 38 countries and submitted in a further 33 countries.
Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
In FIREFISH, 29% (12/41; p<0.0001 compared to natural history) of infants treated with Evrysdi for 12 months were able to sit without support for at least five seconds, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition. This is a key motor milestone never achieved in the natural history of Type 1 SMA. In addition, 93% of infants were alive and 85% were event-free (alive with no permanent ventilation). Furthermore, 5% (2/41) of infants were able to stand with support, as measured by the Hammersmith Infant Neurological Examination, and 83% were able to feed orally. Ninety per cent (37/41) had a CHOP-INTEND* score increase of at least 4 points, with 56% (23/41) achieving a score above 40; the median increase was 20 points. The median age at enrolment was 5.3 months.
In SUNFISH, children and adults treated with Evrysdi experienced a clinically meaningful and statistically significant improvement in motor function at 12 months (1.55 point mean difference; p=0.0156) compared to placebo (1.36 points [95% CI: 0.61, 2.11]; -0.19 points [95% CI: -1.22, 0.84], respectively), as measured by a change from baseline in the Motor Function Measure-32 (MFM-32) total score. Children and adults also experienced significant improvement in upper limb function, a key secondary endpoint, at 12 months (1.59 point mean difference; p=0.0469) compared to placebo (1.61 points [95% CI: 1.00, 2.22]; 0.02 points [95% CI: -0.83, 0.87] respectively), as measured by a change from baseline in the Revised Upper Limb Module (RULM). The median age at enrolment was nine years. Patients treated with Evrysdi for 2-years overall experienced maintenance of improvement in motor function between month 12 and month 24. The mean change from baseline for MFM32 was 1.83 (95% CI: 0.74, 2.92) and for RULM was 2.79 (95% CI: 1.94, 3.64).
Evrysdi demonstrated a favourable efficacy and safety profile, with the safety profile established across the FIREFISH and SUNFISH trials. The most common adverse events were upper respiratory tract infection, pneumonia, nasopharyngitis, pyrexia, constipation, rhinitis, diarrhoea, headache, cough and vomiting. There were no treatment-related safety findings leading to withdrawal from either study.
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. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.
Evrysdi is currently being evaluated in four 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 received other investigational or approved SMA therapies for at least 90 days 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 currently recruiting.
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, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease, Duchenne muscular dystrophy and autism spectrum disorder. 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.
Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.
Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.
Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the twelfth consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).
The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2020 employed more than 100,000 people worldwide. In 2020, Roche invested CHF 12.2 billion in R&D and posted sales of CHF 58.3 billion. 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.
*Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders
All trademarks used or mentioned in this release are protected by law.
Roche Group Media Relations
Phone: +41 61 688 8888 / e-mail: firstname.lastname@example.org
| Dr. Nicolas Dunant|
Phone: +41 61 687 05 17
| Patrick Barth|
Phone: +41 61 688 44 86
| Dr. Daniel Grotzky |
Phone: +41 61 688 31 10
| Karsten Kleine|
Phone: +41 61 682 28 31
| Nina Mählitz|
Phone: +41 79 327 54 74
| Nathalie Meetz|
Phone: +41 61 687 43 05
| Dr. Barbara von Schnurbein|
Phone: +41 61 687 89 67
|Roche Investor Relations|
| Dr. Karl Mahler|
Phone: +41 61 68-78503
| Jon Kaspar Bayard|
Phone: +41 61 68-83894
| Dr. Sabine Borngräber|
Phone: +41 61 68-88027
| Dr. Bruno Eschli|
Phone: +41 61 68-75284
| Dr. Birgit Masjost|
Phone: +41 61 68-84814
| Dr. Gerard Tobin|
Phone: +41 61 68-72942
|Investor Relations North America|
| Loren Kalm|
Phone: +1 650 225 3217
| Dr. Lisa Tuomi|
Phone: +1 650 467 8737
One Liberty Plaza - 165 Broadway
NY 10006 New York
GlobeNewswire 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
Subscribe to all the latest releases from GlobeNewswire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from GlobeNewswire
Skyharbour’s Partner Company Valor Completes Airborne Survey at Hook Lake Uranium Project, Saskatchewan20.4.2021 14:00:00 CEST | Press release
VANCOUVER, British Columbia, April 20, 2021 (GLOBE NEWSWIRE) -- Skyharbour Resources Ltd. (TSX-V: SYH) (OTCQB: SYHBF) (Frankfurt: SC1P) (the “Company”) is pleased to announce that partner company Valor Resources Limited (“Valor”) entered into a contract with Precision GeoSurveys Inc. through TerraLogic Exploration Inc. (“TerraLogic”), the Company’s geological consultants, to undertake a detailed airborne survey over the Hook Lake Project (previously North Falcon Point). The survey commenced in early April 2021 and is now complete. TerraLogic designed the low-level airborne magnetic and VLF-EM survey to cover the entire Hook Lake claim area, using a fixed-wing aircraft, at 75m line-spacing totaling 5,100 line-km. Final data processing, data interpretation, target selection and recommendations for follow-up ground-based exploration and drilling programs will also be handled by TerraLogic. This process is expected to take approximately six weeks, followed immediately by the recommended fi
Acarix publicerar årsredovisningen för 202020.4.2021 13:00:00 CEST | Pressemelding
Pressmeddelande Malmö, 20 april 2021 Acarix publicerar årsredovisningen för 2020 Idag, tisdagen den 20 april, publicerar Acarix sin årsredovisning för verksamhetsåret 2020 på bolagets hemsida www.acarix.com. Årsredovisningen finns även bifogad i pressreleasen. För mer information, vänligen kontakta: Per Persson, vd, e-mail: email@example.com, telefon: +46 73 600 59 90 Informationen lämnades, genom ovanstående kontaktpersons försorg, för offentliggörande vid den tidpunkt som anges av företagets nyhetsdistributör, GlobeNewswire. Om Acarix Acarix grundades 2009 och är noterat på Nasdaq First North Premier Growth Market (ticker: ACARIX). Acarix CADScor®System använder en avancerad sensor som placeras på huden ovanför hjärtat för att lyssna på ljudet av hjärtkontraktion och turbulent flöde. Det har utformats för att vara ett allt-i-ett-system i den meningen att signalen från hjärtat spelas in, bearbetas och visas som en patientspecifik s k CAD-score på skärmen. Presentation av mätresul
Acarix publishes the annual report for 202020.4.2021 13:00:00 CEST | Press release
Press release Malmö, Sweden, April 20, 2021 Acarix publishes the annual report for 2020 Today, Tuesday April 20, Acarix is publishing its Annual Report for 2020 on the company website www.acarix.com. The Annual Report is also attached to this press release. For further information, please contact: Per Persson, CEO, E-mail: firstname.lastname@example.org, Phone: +46 73 600 59 90 The information was provided, through the agency of the above contact person, for publication at the time specified by the company's news distributor, GlobeNewswire. About Acarix: Acarix was established in 2009 and is listed on Nasdaq First North Premier Growth Market (ticker: ACARIX). Acarix’s CADScor®System uses an advanced sensor placed on the skin above the heart to listen to the sounds of cardiac contraction movement and turbulent flow. It has been designed to be an all-in-one system in the sense that the heart signal will be recorded, processed, and displayed as a patient specific score, the CAD-score, on the devi
GW Pharmaceuticals receives European Commission approval for EPIDYOLEX® (cannabidiol) for the treatment of seizures associated with tuberous sclerosis complex20.4.2021 12:30:00 CEST | Press release
– Tuberous sclerosis complex (TSC) represents a third indication for GW’s cannabidiol in Europe – – TSC causes epilepsy in up to 85% of patients, and up to 60% of those patients do not respond to standard anti-epileptic medicines1,2,3– LONDON, April 20, 2021 (GLOBE NEWSWIRE) -- GW Pharmaceuticals plc (Nasdaq: GWPH) (“GW”, “the Company” or “the Group”), a world leader in discovering, developing and delivering regulatory approved cannabis-based medicines, today announces that the European Commission (EC) has approved the Type II variation application for EPIDYOLEX® (cannabidiol) as an adjunctive treatment of seizures associated with TSC, for patients two years of age and older. This approval, which represents the third indication for GW’s cannabidiol in Europe, paves the way for the expanded launch of the medicine across Europe. “This is an exciting moment for patients, and their families, who are affected by this debilitating disease and may benefit from this treatment. This approval is
Fingerprint Cards AB: invitation to the first quarter 2021 results presentation20.4.2021 11:30:00 CEST | Press release
Fingerprint Cards AB (Fingerprints™) will host a telephone conference and webcast presentation of its first quarter 2021 results, on April21, 2021 at 09:00 CEST. Fingerprints’ CEO Christian Fredrikson will present the report together with CFO Per Sundqvist in a combined webcast and telephone conference. The presentation will be held in English. Time: Wednesday, April 21, 2021 at 09:00 am CEST. Location: combined webcast and telephone conference. The report is available at https://www.fingerprints.com/ The webcast and the presentation material can be accessed through https://edge.media-server.com/mmc/p/5dpwm66j where it is also possible to ask questions. For media and analysts: The telephone conference dial-in is +44 (0) 2071 928000 (international participants) or 08-506 921 80 (Swedish participants). Please state conference ID 9862978. About Fingerprints Fingerprint Cards AB (Fingerprints) – the world’s leading biometrics company, with its roots in Sweden. We believe in a secure and se
Fingerprint Cards AB: inbjudan till presentation av resultatet för första kvartalet 202120.4.2021 11:30:00 CEST | Pressemelding
FingerprintCardsAB (Fingerprints™)bjuder in till telefonkonferens och en webbsänd presentation av resultatet för första kvartalet 2021, den 21april 2021 klockan 09:00. Fingerprints vd Christian Fredrikson presenterar rapporten tillsammans med CFO Per Sundqvist i en kombinerad webbsändning och telefonkonferens. Presentationen sker på engelska. Tid: onsdagen den 21 april 2021, klockan 09:00 CEST. Plats: kombinerad webbsändning och telefonkonferens. Rapporten finns på https://www.fingerprints.com/ Webbsändningen och presentationsmaterialet finns på https://edge.media-server.com/mmc/p/5dpwm66j där man även har möjlighet att ställa frågor. För media och analytiker: Telekonferensen nås via +44 (0) 2071 928000 (internationella deltagare) eller 08-506 921 80 (svenska deltagare). V.g. uppge konferens-ID 9862978. Om Fingerprints Fingerprint Cards AB (Fingerprints) - det ledande globala biometribolaget med svenska rötter. Vi tror på en säker och sammanbunden värld, där du är nyckeln till allt. Vå
RESULT OF RIKSBANK REVERSED AUCTIONS KOMMUNINVEST BONDS20.4.2021 11:10:00 CEST | Press release
RESULT OF RIKSBANK REVERSED AUCTIONS KOMMUNINVEST BONDS Auction date2021-04-20Loan2410 Coupon1.00 %ISIN-codeSE0010469205Maturity2024-10-02 Tendered volume, SEK mln500 +/- 250 Volume offered, SEK mln2,250Volume bought, SEK mln750 Number of bids8 Number of accepted bids2 Average yield0.088 %Lowest accepted yield0.087 %Highest yield0.088 %% accepted at lowest yield 50.00 Auction date2021-04-20Loan2602 Coupon0.75 %ISIN-codeSE0013745452Maturity2026-02-04 Tendered volume, SEK mln500 +/- 250 Volume offered, SEK mln2,000 Volume bought, SEK mln500 Number of bids5 Number of accepted bids1 Average yield0.288 %Lowest accepted yield0.288 %Highest yield0.288 %% accepted at lowest yield 100.00 Auction date2021-04-20LoanG2306Coupon0.625 %ISIN-codeXS1897258098Maturity2023-06-01 Tendered volume, SEK mln250 +/- 250 Volume offered, SEK mln0 Volume bought, SEK mln0 Number of bids0 Number of accepted bids0 Average yieldNALowest accepted yieldNAHighest yieldNA% accepted at lowest yield NA