DBV Technologies SA blindsided investors by disclosing that the U.S. FDA had placed a partial clinical hold on its phase III Vitesse trial of its Viaskin Peanut patch immunotherapy for peanut allergy, calling for several changes to the study protocol. Its demands came just two weeks after the company claimed to have finalized the protocol in consultation with the agency.
Although it wasn’t a shutout, Spectrum Pharmaceuticals Inc. didn’t get the support it needed from a U.S. FDA advisory committee Sept. 22 for its non-small-cell lung cancer candidate, poziotinib, which it has proposed marketing as Pozenveo.
It took resolving issues from two complete response letters for Fennec Pharmaceuticals Inc. to finally land U.S. FDA approval of Pedmark. The injectable formulation of sodium thiosulfate is for reducing the risk of platinum-induced ototoxicity associated with cisplatin in pediatric patients 1 month and older with localized, non-metastatic solid tumors. Approval of the NDA arrived Sept. 20, three days before its Sept. 23 PDUFA date.
The fate of three cancer drugs, and possibly the future financial health of their sponsors, could be on the line Sept. 22 and 23 as the U.S. FDA’s Oncologic Drugs Advisory Committee (ODAC) takes a hard look at the safety-efficacy data for Spectrum Pharmaceuticals Inc.’s Pozenveo, Oncopeptides AB’s Pepaxto and Secura Bio Inc.’s Copiktra. First up in the triple-header is Pozenveo (poziotinib), which is seeking accelerated approval as a second-line treatment for patients with locally advanced or metastatic non-small-cell lung cancer harboring HER2 exon 20 insertion mutations confirmed by an FDA-approved test.
Claiming it would be impossible to carve out a so-called skinny label that would comply with generic drug “same labeling” rules, Novartis AG is petitioning the U.S. FDA, for the second time, not to approve generic versions of its blockbuster heart drug, Entresto (sacubitril + valsartan), that attempt to carve around a cardiovascular indication that has exclusivity until Feb. 16, 2024.
Bluebird Bio Inc.’s elivaldogene autotemcel (eli-cel) gained U.S. approval late Sept. 16 for use in early active cerebral adrenoleukodystrophy (CALD), making it the firm’s second gene therapy to clear the FDA in as many months. Branded Skysona, eli-cel is expected to be available commercially by the end of 2022 and its launch will require only “incremental” company resources on top of those required for the ongoing launch of beta-thalassemia gene therapy Zynteglo (betibeglogene autotemcel), Bluebird said.
With four complete response letters behind it and seven months ahead of its April 2023 PDUFA date, Mallinckrodt plc’s Terlivaz (terlipressin) has been approved by the U.S. FDA for treating hepatorenal syndrome. Terlivaz had a decade-long series of obstacles before getting the long-awaited approval. The vasopressin analogue selective for V1 receptors was approved, in part, on results from the phase III CONFIRM trial of 300 patients, which met its primary endpoint of renal function improvement, avoidance of dialysis and short-term survival (p=0.012).
From the beginning of the monkeypox outbreak in the U.S. in May, the federal government has bungled the response, according to both Democratic and Republican members of the Senate Health, Education, Labor and Pensions (HELP) Committee.
Akouos Inc. has received IND clearance from the FDA to initiate a first-in-human phase I/II pediatric trial of AK-OTOF, a gene therapy intended for the treatment of patients with otoferlin gene (OTOF)-mediated hearing loss.
Transcenta Holding Ltd. has received IND clearance from the FDA for TST-003, its first-in-class, high-affinity, humanized monoclonal antibody targeting gremlin-1.
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