— Sarepta will seek re-examination of the opinion and request that a Scientific Advisory Group (SAG) be convened —
CAMBRIDGE, MA, USA I June 01, 2018 I Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), has adopted a negative opinion for EXONDYS® (eteplirsen), as expected. EXONDYS is designed to treat patients with Duchenne muscular dystrophy (DMD) amenable to skipping exon 51 of the dystrophin gene.
As previously reported on Sarepta’s May 5, 2018 earnings call, a negative opinion was anticipated following the oral explanation trend vote. Sarepta will request a re-examination of the opinion, which will result in the assignment of a new rapporteur and co-rapporteur. The Company will also request a Scientific Advisory Group (SAG) on DMD be called so that neuromuscular specialists, experienced with working with treatments for these patients, can provide expert guidance and insight into, among other things, the validity of the external controls used and the importance of certain functional endpoints, including for instance, the relevance of meaningful slowing pulmonary decline in patients with this difficult to treat disease. The re-examination process is expected to be completed by year-end 2018.
About Duchenne Muscular Dystrophy
DMD is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 – 5,000 male births worldwide.
About EXONDYS® (eteplirsen)
EXONDYS® (eteplirsen) is being developed to treat individuals who have a specific type of Duchenne muscular dystrophy (DMD), comprising 13 percent of the overall population who have one of the mutations amenable to exon 51 skipping. If approved, it would be the first injectable therapy in Europe designed to treat the underlying cause of DMD — the lack of a protein called dystrophin, which is critical for muscles to function properly.
EXONDYS utilizes Sarepta’s precision RNA splicing technology to correct specific genetic mutations and produce a shortened, but functional, form of the dystrophin protein.
About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates. For more information, please visit www.sarepta.com.
SOURCE: Sarepta Therapeutics
Post Views: 22
— Sarepta will seek re-examination of the opinion and request that a Scientific Advisory Group (SAG) be convened —
CAMBRIDGE, MA, USA I June 01, 2018 I Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), has adopted a negative opinion for EXONDYS® (eteplirsen), as expected. EXONDYS is designed to treat patients with Duchenne muscular dystrophy (DMD) amenable to skipping exon 51 of the dystrophin gene.
As previously reported on Sarepta’s May 5, 2018 earnings call, a negative opinion was anticipated following the oral explanation trend vote. Sarepta will request a re-examination of the opinion, which will result in the assignment of a new rapporteur and co-rapporteur. The Company will also request a Scientific Advisory Group (SAG) on DMD be called so that neuromuscular specialists, experienced with working with treatments for these patients, can provide expert guidance and insight into, among other things, the validity of the external controls used and the importance of certain functional endpoints, including for instance, the relevance of meaningful slowing pulmonary decline in patients with this difficult to treat disease. The re-examination process is expected to be completed by year-end 2018.
About Duchenne Muscular Dystrophy
DMD is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 – 5,000 male births worldwide.
About EXONDYS® (eteplirsen)
EXONDYS® (eteplirsen) is being developed to treat individuals who have a specific type of Duchenne muscular dystrophy (DMD), comprising 13 percent of the overall population who have one of the mutations amenable to exon 51 skipping. If approved, it would be the first injectable therapy in Europe designed to treat the underlying cause of DMD — the lack of a protein called dystrophin, which is critical for muscles to function properly.
EXONDYS utilizes Sarepta’s precision RNA splicing technology to correct specific genetic mutations and produce a shortened, but functional, form of the dystrophin protein.
About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates. For more information, please visit www.sarepta.com.
SOURCE: Sarepta Therapeutics
Post Views: 22
