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Press Release: Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA)

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U.S. Food and Drug Administration (FDA) extends review time for risdiplam
following agreed submission of additional data, including SUNFISH Part 2

Roche has submitted filing applications for risdiplam in seven countries
with submission in China imminent

Risdiplam is being investigated in infants, children and adults with Type
1, 2 or 3 SMA

Basel, 07 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY), today
announced that the U.S. Food and Drug Administration (FDA) has extended
the Prescription Drug User Fee Act (PDUFA) date for its review of the
New Drug Application (NDA) of risdiplam with a decision expected by
August 24, 2020. The extension is a result of the recent submission of
additional data by Roche, including data from the pivotal SUNFISH Part 2
study, in close collaboration with the FDA. These data were recently
presented at the 2nd International Scientific and Clinical Congress on
Spinal Muscular Atrophy.



In November 2019, the FDA granted Priority Review for risdiplam with a
decision for approval expected by May 24, 2020. In February 2020, based
on discussions with the FDA, Roche submitted additional data which could
help ensure access to risdiplam for a broad range of people living with
the condition, if approved. This included 12-month efficacy and safety
data from the pivotal SUNFISH Part 2 study (n=180), the only
placebo-controlled study ever undertaken in people aged 2-25 years with
Type 2 or 3 SMA. Given the volume of additional data submitted, the FDA
requires more time for review.



"We strongly believe in the potential of risdiplam as a new therapeutic
option and recognize that unmet need remains in the treatment of SMA,"
said Levi Garraway, M.D., Ph.D., chief medical officer and head of
Global Product Development. "We are working closely with the FDA to
support the review of risdiplam. Our goal is to bring this therapy to
infants, children and adults living with SMA as quickly as possible."



As part of our continued commitment to people living with SMA, Roche is
pleased to announce that we have also submitted in Brazil, Chile,
Indonesia, Russia, South Korea and Taiwan. Filing in China is imminent
and we are currently on track to submit a Marketing Authorization
Application (MAA) to the European Medicines Agency in mid-2020 as well
as other international markets.



Roche leads the clinical development of risdiplam, an investigational,
orally administered survival motor neuron-2 (SMN2) splicing modifier for
SMA, as part of a collaboration with the SMA Foundation and PTC
Therapeutics. Risdiplam is being studied in a broad clinical trial
programme in SMA, with patients ranging from birth to 60 years old, and
includes patients previously treated with other SMA-targeting therapies.
The clinical trial population is designed to represent the broad,
real-world spectrum of people living with this disease with the aim of
ensuring access for all appropriate patients.

About 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 babies. 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 risdiplam

Risdiplam is an investigational survival motor neuron-2 (SMN2) splicing
modifier for SMA and is an orally administered liquid. It is designed to
durably increase and sustain SMN protein levels both throughout the
central nervous system and in peripheral tissues of the body. It is
being evaluated for its potential ability to help the SMN2 gene produce
more functional SMN protein throughout the body.



Risdiplam is currently being evaluated in four multicentre trials in
people with SMA:


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 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.

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. The primary objective of Part 1 was to assess the safety profile
of risdiplam in infants and determine the dose for Part 2. Part 2 is a
pivotal, single-arm study of risdiplam in 41 infants with Type 1 SMA
treated for 24 months, followed by an open-label extension. Enrolment for
Part 2 was completed in November 2018. The primary objective of Part 2 is
to assess efficacy as measured by the proportion of infants sitting
without support after 12 months of treatment, as assessed in 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).

JEWELFISH (NCT03032172) an open-label exploratory trial in people with
SMA aged 6 months60 years who have been previously treated with
SMA-directed therapies. The study has completed recruitment.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.

About Roche in neuroscience

Neuroscience is a major focus of research and development at Roche. The
company's goal is to develop treatment options based on the biology of
the nervous system to help improve the lives of people with chronic and
potentially devastating diseases.



Roche has more than a dozen investigational medicines in clinical
development for diseases that include multiple sclerosis, spinal
muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer's
disease, Huntington's disease, Parkinson's disease, Duchenne muscular
dystrophy and autism.



About Roche

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
eleventh 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 2019 employed about 98,000 people worldwide. In
2019, Roche invested CHF 11.7 billion in R&D and posted sales of CHF
61.5 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
https://www.globenewswire.com/Tracker?data=rt3DGVxXN_SNqMCtmpOgy4DzplPhqtM7u09EvgDCmE8JyNJhQDfhIPHpUgtbahhXRT0GDDw3z18kptzdTyBTwQ
www.roche.com.



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







Roche Group Media Relations

Phone: +41 61 688 8888 / e-mail:
https://www.globenewswire.com/Tracker?data=AidJWA0ljYRAMi_NckSgz75Nza_egNMdpArpW5-zLfs3rWcs_tNZ_VoKAAqHWGPloNSUHbzjOhukAYCNjLDGgLfAVj4AVhJjFzENnc9hHzihKCk7nLAPLj3hgIph0aaP
media.relations@roche.com

- Nicolas Dunant (Head)

- Patrick Barth

- Daniel Grotzky

- Karsten Kleine

- Nathalie Meetz

- Barbara von Schnurbein

Attachment


Roche_mediarelease_risdiplam_07042020
https://ml-eu.globenewswire.com/Resource/Download/0220d09e-90e8-4db7-a1aa-1e8f6514af61








(END) Dow Jones Newswires

April 07, 2020 16:05 ET ( 20:05 GMT)
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