FDA News Roundup: Pfizer, Novartis, Sanofi, and More
By Anna Rose Welch, Editorial & Community Director, Advancing RNA
What drug approvals have you missed recently?
Celgene Granted Indication Expansion For Revlimid + Dexamethasone
The FDA gave Celgene’s Revlimid + dexamethasone the nod to be used in patients that are newly diagnosed with multiple myeloma. The drug combo has been out on the market since 2006 when the FDA approved it for those with multiple myeloma who had taken at least one previous treatment. The Revlimid/dexamethasone combination was investigated in several phase 3 studies, including the FIRST trial, which determined treatment with the combo significantly extended progression free survival compared to melphalan, prednisone, and thalidomide (MPT).
Pfizer Rapamune Accepted For Priority Review
Pfizer’s sNDA for Rapamune, its immunosuppressant for the rare lung disease lymphangioleiomyomatosis, was accepted for priority review, with an expected action date in June. The drug proved its ability to stabilize lung function in a trial that enrolled 89 LAM patients. Should the drug be approved, Rapamune would hold the record for being the first treatment option for those suffering from LAM.
Novartis' Farydak Approved For Multiple Myeloma
Novartis’ HDAC inhibitor, Farydak, was approved in combination with bortezomib and dexamethasone for multiple myeloma patients who had not seen improvements with two prior treatments (including bortezomib and an immunomodulatory agent). In the PANORAMA-1 trial, the drug was found to extend progression-free survival compared to placebo. This is the first HDAC inhibitor to hit the market for those with multiple myeloma, and the drug is expected to restore cell function in multiple myeloma because of its epigenetic abilities.
FDA Accepts Synageva’s BLA For Priority Review
The FDA accepted Synageva BioPharma’s Biologics License Application (BLA) for Kanuma, a recombinant form of human LAL enzyme, indicated for lyosomal acid lipase deficiency (LAL deficiency). The company expects a decision by September. The application includes data from a Phase 3 trial that investigated the drug in children and adults with LAL deficiency. The treatment was also tested in infants with LAL deficiency in a phase2/3 clinical trial.
FDA Approves New Antibiotic From Actavis
Actavis got a nod of approval from the FDA for a new antibiotic, Avycaz, which can be used to treat complicated intra-abdominal infections and complicated urinary tract infections. The drug is a combination of ceftazidime and avibactam, a new beta-lactamase inhibitor. The drug was previously designated a Qualified Infectious Disease Product (QIDP), making it the fifth drug with this designation to earn approval. Avycaz underwent investigation in two Phase 2 trials — one for each indication.
Sanofi Wins FDA Nod For Diabetes Drug
The FDA gave Sanofi the green light for its Toujeo diabetes drug, which is intended to be a stronger replacement for the company’s blockbuster Lantus as it loses patent protection this month. Toujeo contains insulin glargine, which is the same active ingredient as Lantus, however it is present at three times the concentration and is released more slowly. The drug was approved to treat those with both type 1 and type 2 diabetes after demonstrating its comparability to Lantus in the EDITION clinical trial program. While it proved capable of maintaining the same level of control over blood sugar levels as Lantus, the drug does come with a warning that it can cause severe symptomatic hypoglycemia.
Insys Therapeutics' Cannabidiol Put On Fast Track
Last week, the FDA granted Fast-Track Designation to Insys Therapeutics’ cannabidiol indicated for Dravet syndrome. The treatment previously earned orphan drug designation in Dravet syndrome. Now that the company has earned a thumbs up from the FDA, it will be launching a clinical trial program for the drug later in the year. This program will include a Phase 1 pharmacokinetic study in epilepsy patients, along with a Phase 3 for those with Dravet syndrome and the equally rare Lennox Gastaut syndrome—an indication for which the drug previously earned orphan drug designation as well.