Congress has voted to extend the FAA’s tax authority for six months. As anticipated, the extension did not include any of the disability provisions set out in the full bill, but the possibility of these provisions being included in the full reauthorization in March remains.
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.
“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.