Device makers have reporting responsibilities for devices used in combination products per an FDA final rule published in 2016, but the FDA said in the 2018 draft guidance that the applicant of a drug- or biologic-led combo product must evaluate whether a malfunction of the device component would suggest a hazard for similar combinations using that device. That provision appears in the final rule, and thus puts the onus on makers of drugs and biologics to do a job device makers say is theirs to handle.

Following more than a year of restructuring and recalibration, Regulus Therapeutics Inc. is facing a new partial clinical hold placed by the FDA on its phase I test of RGLS-4326, an oligonucleotide it's developing for the treatment of autosomal dominant polycystic kidney disease (ADPKD). 

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.

The FDA placed a clinical hold Wednesday on a phase I trial by Unum Therapeutics Inc. after a patient experienced serious adverse events that included grade 3 neurotoxicity and cytomegalovirus infection, and grade 4 respiratory distress.

With regulatory science always lagging innovation, ambiguity has long been a certainty at the FDA and is likely to become even more so as the pace of new technologies quickens.

In the words of former FDA Commissioner Scott Gottlieb, the five-week government shutdown that ended Jan. 25 was "the most difficult operational challenge we have faced in modern times." While the full impact of that challenge could ripple through the FDA for a while, it made little difference in the number of warning letters the agency sent out between Dec. 23 and Jan. 25.

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.