Tag Archive for: New Drug Application

After VX-548 demonstrated significant pain relief in surgical and non-surgical settings, Vertex Pharmaceuticals is preparing to file a New Drug Application for the non-opioid candidate by mid-2024.

In a watershed U.S. regulatory milestone, the MAPS Public Benefit Corporation announced Tuesday that it has submitted a New Drug Application to the FDA for an investigational MDMA-assisted therapy for individuals with post-traumatic stress disorder.

Aldeyra Therapeutics is planning to run another trial, in line with the regulator’s requirements, and is expecting top-line data and a New Drug Application resubmission in the first half of 2024.

In a late-stage study, most Huntington’s disease patients reported that their symptoms of chorea were “much improved” or “very much improved” following treatment with Neurocrine Biosciences’ VMAT2 inhibitor Ingrezza.

The label for the achondroplasia drug, which promotes endochondral bone growth, now covers children under five years of age with the rare genetic disease causing the most common form of dwarfism.

This follows a late-cycle review meeting in which the regulator flagged “substantive review issues” with the New Drug Application (NDA). The FDA pointed out to Aldeyra that it does not appear to “have data to support the clinical relevance of the ocular signs to support your dry eye indication,” according to the company’s SEC filing.

The FDA will finish September with three action dates and one of the year’s most highly-anticipated advisory committee meetings.

This week, GSK is expecting a verdict for its myelofibrosis candidate momelotinib and Alnylam will make the case for patisiran in cardiomyopathy of ATTR amyloidosis.

Akebia Therapeutics scored a long-awaited win Tuesday when it announced the FDA has granted a path forward for vadadustat in anemia associated with chronic kidney disease in dialysis-dependent patients, which will not require the company to submit new data. 

Findings from the ACTION-Galactosemia Kids study showed Applied Therapeutics’ govorestat fell short of its primary endpoint, inducing no significant functional improvement in children with galactosemia, a rare genetic metabolic disease.