Last week was a rare case where the world’s leading drugs and devices regulators were all in action simultaneously. The US Food and Drug Administration (FDA), the European Medicines Agency (EMA) and China’s National Medical Products Administration (NMPA) doled out the following rulings and recommendations with the FDA disseminating most of the good and bad news to aspiring manufacturers.
Celgene Corp. (Summit NJ) announced Friday that the FDA has expanded the label for the PDE4 inhibitor Otezla (apremilast) to include treatment of oral ulcers in adults with Behçet’s disease. Approval was based on findings from the Phase 3 RELIEF trial evaluating Otezla in 207 Behçet’s disease patients with active oral ulcers who were previously treated with at least one non-biologic medication and were candidates for systemic therapy. Results demonstrated that Otezla-treated patients achieved a 42.7-point reduction from baseline in the pain of oral ulcers as measured using a visual analogue scale at week 12, compared with an 18.7-point reduction for placebo.
Elsewhere, Eli Lilly & Co. (Indianapolis IN) announced that the FDA approved Baqsimi (glucagon) for the treatment of severe hypoglycemia in people with diabetes ages four years and above, making the drug the first nasally administered formulation of rescue glucagon authorized in the US. The company noted that the product is expected to be available in pharmacies within one month and will carry a list price of $280.80 for a one-pack and $561.60 for a two-pack. Baqsimi is a portable, dry nasal spray form of glucagon, ready to use with no reconstitution or priming required in a single, fixed 3mg dose.
The FDA has approved multiple applications for the first generics of pregabalin (Lyrica) from Pfizer Inc. (NYC) for the management of neuropathic pain. The FDA granted approvals for the generic versions of Lyrica to Alembic Pharmaceuticals, Alkem Laboratories, Amneal Pharmaceuticals, Dr. Reddy’s Laboratories, InvaGen Pharmaceuticals, MSN Laboratories, Rising Pharmaceuticals, Sciegen Pharmaceuticals, and Teva Pharmaceuticals. Among other indications, Pregabalin is indicated for neuropathic pain related to diabetic neuropathy, postherpetic neuralgia, and spinal cord injury.
The FDA announced the approval of Pfizer Inc.’s (NYC) Ruxience (rituximab-pvvr) as a biosimilar referencing Roche and Biogen’s Rituxan (rituximab) for the treatment of adults with CD20-positive B-cell non-Hodgkin’s lymphoma (NHL) to be used as a single agent or in combination with chemotherapy, as well as for adults with CD20-positive chronic lymphocytic leukemia in combination with chemotherapy. The agency added that Ruxience is also the first biosimilar approved to treat granulomatosis with polyangiitis and microscopic polyangiitis.
Samsung Bioepis (Seoul KOR) announced that the FDA approved Hadlima (adalimumab-bwwd), a biosimilar version referencing AbbVie’s Humira (adalimumab), for the treatment of adult Crohn’s disease, ankylosing spondylitis, juvenile idiopathic arthritis, psoriatic arthritis, plaque psoriasis, rheumatoid arthritis and ulcerative colitis. The drugmaker noted that the therapy, which will be marketed by Merck & Co. in the US, will be launched in the country after June 30, 2023 under the terms of a patent infringement lawsuit settlement with AbbVie.
Biohaven Pharmaceutical Holding Co. Ltd. (New Haven CT) announced that it has received a complete response letter from the FDA for its marketing application seeking approval for Nurtec to treat patients with amyotrophic lateral sclerosis (ALS). Nurtec, also known as BHV-0223, is a sublingual formulation of riluzole, which is already FDA-approved for ALS, but currently only exists as an oral tablet taken with water or as an oral suspension.
For Pfizer Inc.’s (NYC) blockbuster immunology drug Xeljanz, an FDA safety communication has now turned into a boxed warning. And forget the drug’s approval as a first-line ulcerative colitis treatment, at least for now. In a new addition to Xeljanz’s label on Friday, the FDA is warning of an increased risk of blood clots–and potentially, death–with the JAK inhibitor’s 10-mg twice daily dose. And meanwhile, the drug’s green light in ulcerative colitis has been postponed.
And the FDA has sent a warning letter to Curaleaf Inc. (Wakefield MA) for illegally selling unapproved products containing cannabidiol (CBD) online with unsubstantiated claims that the products treat cancer, Alzheimer’s disease, opioid withdrawal, pain, and other conditions or diseases.
Across the Pond, in a busy meeting last week, experts with the European Medicines Agency’s Committee for Medicinal Products for Human Use endorsed four new drugs, a Mylan NV generic, and numerous new indications for already-marketed meds.
Recommendations for approval:
GW Pharmaceuticals PLC’s (London) Epidyolex, the closely watched cannabis-based drug to treat seizures associated with Lennox-Gastaut syndrome or Dravet syndrome, now looks set for its European nod. The drug, sold as Epidiolex in the US, won CHMP backing and now awaits a final EU decision within two months, GW said.
Acorda Therapeutics Inc. (Ardsley NY), which suffered a big patent loss for Ampyra in the US last year, looks set to roll with Inbrija in Europe in the coming months. CHMP officials endorsed the med to treat symptoms of “off” periods for patients with Parkinson’s disease. Last year, Acorda lost its Ampyra patent protection in the US, forcing it to cut costs and jobs and switch gears to focus on the newer med.
Bayer AG’s (Leverkusen DEU) Vitrakvi, a “tumor-agnostic” drug that targets solid tumors with the NTRK gene fusion, scored a CHMP recommendation for conditional marketing approval. In the US, the drug scored an approval in November 2018.
Canada’s Theratechnologies Inc. scored an endorsement for Trogarzo, an HIV drug with a novel mechanism of action. Trogarzo won US FDA approval last March.
All the recommendations will go to the European Commission for final approval.
Finally, Novartis AG (Basel CHE) announced Friday that China’s National Medical Products Administration (NMPA) approved Gilenya (fingolimod) for relapsing forms of multiple sclerosis for adults, as well as for children aged 10 years and older.