PTC Therapeutics reported today the U.S. Food and Drug Administration’s Office of Drug Evaluation is unable to approve the company’s New Drug Application for ataluren (brand name Translarna) for the treatment of Duchenne muscular dystrophy (DMD) caused by a premature stop codon, or nonsense mutation, in its current form.
FDA: Soliris is approved to treat adults with generalized MG.
The drug therapy ATB200/AT2221, meant to treat Pompe disease, has received FDA Orphan Drug Designation.
“It truly feels as if my entire career path has been leading me to this role,” said Vos. “I am excited to bring my passion for patient care and advocacy, as well as my success using new technology to build new, innovative communication approaches, to lead MDA in the fast-changing world of research and patient care.”
The FDA yesterday convened a meeting with the PCNS, to review the NDA, for ataluren (brand name Translarna) to treat some forms of DMD. Following a presentation of the data in support of the NDA by PTC Therapeutics the overwhelming majority (10 out of 11) suggested that although it is possible that Translarna may be effective, the data are inconclusive and more work is needed to determine whether, in fact, it is.
The FDA on Thursday will convene a meeting with the Peripheral and Central Nervous Systems Drugs Advisory Committee (PCNS), to review the new drug application, or NDA, for ataluren (brand name Translarna) DMD.
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Just in time for back to school: updated guides for teachers of students living and learning with neuromuscular diseases
The Senate will soon begin consideration of the S. 1519, National Defense Authorization Act (NDAA), which includes provisions that severely restrict, and in some cases terminate, critical medical research. Please join us in taking action and urging your Senators to support the bipartisan amendment to the National Defense Authorization Act that will ensure funding for biomedical research continues.
State specific Hurricane Irma resources for Florida, Georgia, North Carolina and Virginia.
