While a significant number of clinical trial delays occurred during the month of April, it was business as usual from a regulatory standpoint for companies with late-stage therapies ready for the market and for those targeting underserved patient populations.
It's been a busy September for Ardelyx Inc. so far. Less than two weeks after the Fremont, Calif-based company reported promising pivotal data for tenapanor in chronic kidney disease patients with hyperphosphatemia, the sodium hydrogen exchanger 2 (NHE3) inhibitor won FDA approval for use in irritable bowel syndrome with constipation (IBS-C).
After two complete response letters (CRLs), Agile Therapeutics Inc. with its Twirla (levonorgestrel/ethinyl estradiol) contraceptive patch – hounded by FDA concerns regarding manufacture and adhesion properties – might just be on the road to success.
Despite a February adcom meeting urging the agency to wait for more data, the FDA has approved Karyopharm Therapeutics Inc.'s selinexor, in combination with dexamethasone (dex), as a new treatment for certain adults with relapsed refractory multiple myeloma (MM). The approval covers patients who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents and an anti-CD38 monoclonal antibody.
Approval of Alexion Pharmaceutical Inc.'s Soliris (eculizumab) injection to treat neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive makes it the first and only FDA-approved treatment. It's also one of the most expensive treatments in the world, making it vulnerable to off-label use and the eventual creep of biosimilars into the market.