FDA Decision Tracker 2023: BioMarin, CellTrans, Pfizer and OPKO Health

 The FDA has been on a roll in the first half of 2023, approving 28 novel treatments thus far. And if the first six months is any indication, the biopharma industry should expect several more novel therapies to be greenlit, including a few more first-in-class treatments. But it’s not always good news that companies have to deliver to their stakeholders; the year has already seen its fair share of Complete Response Letters.

BioSpace looks forward to keeping you up to date on all the FDA’s action as we launch our FDA Decision Tracker.  

June

June 29:

Capping a busy week, the FDA approved BioMarin’s Roctavian (valoctocogene roxaparvovec-rvox) as the first gene therapy for adults with severe hemophilia A. Hemophilia is a rare genetic bleeding disorder caused by a mutation in the gene that encodes factor VIII (FVIII), which is necessary for blood to clot. A one-time gene therapy, Roctavian contains a healthy gene for factor VIII. Delivered through an adeno-associated virus (AAV) vector, the gene is expressed in the liver to increase blood levels of FVIII, thereby reducing the risk of uncontrolled bleeding. 

June 28:

The FDA notched another milestone with the approval of the first cellular therapy for type 1 diabetes. Lantidra (donislecel) developed by Chicago-based CellTrans, is a cell therapy made from the pancreatic islet cells of deceased donors. It is intended for adult patients whose repeated hypoglycemic episodes leave them unable to hit average blood glucose levels. In a clinical trial of 30 patients, 21 were insulin-free for at least a year; 11 didn’t require insulin for between one and five years and 10 were insulin-free for more than five years. Five patients failed to achieve any days of insulin independence.

June 28:

Pfizer’s bet on OPKO Health’s human growth hormone analog paid off as the FDA approved the treatment—which will be marketed as Ngenla—to treat children whose production of growth hormones is impaired. Pfizer purchased exclusive global commercialization rights for the then-experimental treatment in December 2014 for $295 million upfront and a promise of up to $275 million in milestones. This was the partners’ second try for FDA approval after the regulator rejected Ngenla’s first bid in January 2022. Pfizer and OPKO did not state the reasons for the initial denial.

June 28:

The FDA declined to approve Eton Pharmaceuticals’dehydrated alcohol injection for the treatment of methanol poisoning, citing concerns related “primarily to Chemistry Manufacturing and Controls.” This is the second Complete Response Letter issued to Eton for this proposed treatment. In the first, issued in March 2021, the FDA indicated that travel restrictions due to the COVID-19 pandemic prevented a timely inspection of the company’s European contract manufacturing site.

June 27:

The FDA approved UCB’s Rystiggo (rozanolixizumab), a subcutaneously administered humanized IgG4 monoclonal antibody, to treat generalized myasthenia gravis, a rare muscle-wasting autoimmune disease. Rystiggo is the only treatment approved to treat patients who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive. The regulatory green light was based on a Phase III trial that showed Rystiggo led to significant improvements in symptoms related to breathing, talking, swallowing and rising from a chair.

June 23:

The FDA greenlit Pfizer’s Litfulo (ritlecitinib) for the treatment of patients as young as 12 years with severe alopecia areata, an autoimmune disease characterized by patchy or complete hair loss on the scalp, face or body. Litfulo inhibits Janus kinase 3 and the tyrosine kinase and is believed to work by blocking the signaling of cytokines and cytolytic activity of T cells, which are implicated in alopecia areata.

June 22: 

In one of the year’s most-anticipated regulatory decisions, the FDA approved Sarepta’s Elevidys—formerly SRP-9001—for children 4 to 5 years with Duchenne muscular dystrophy. Elevidys is the first-ever gene therapy for DMD, a neuromuscular disease characterized by progressive muscle weakness and atrophy that strikes primarily young boys. The gene therapy was approved via the FDA’s accelerated approval pathway based on data that established it increased the expression of the Elevidys micro-dystrophin protein.

June 21:

Eli Lilly and Boehringer Ingleheim’s Jardiance (empagliflozin) and Synjardy (empagliflozin and metformin hydrochloride) picked up another indication, becoming the first and only SGLT2 inhibitors approved for children 10 years and older with type 2 diabetes. Previously, there had only been one oral drug for this indication: metformin, which was approved in 2000.

June 20:

Argenx will bring to market the first subcutaneous injectable for generalized myasthenia gravis (gMG), a rare muscle-wasting autoimmune disease. Vyvgart (efgartigimod) was first approved in 2021 as a one-hour intravenous infusion. Vyvgart is an antibody fragment that binds to the neonatal Fc receptor to prevent recycling of immunoglobulin G back into the blood. This includes a reduction in abnormal AChR antibodies, which are present in approximately 85% of gMG patients. AChR antibodies block the acetylcholine receptors from being able to receive signals from the nerve to stimulate a muscular response. Their normalization should ostensibly improve muscular function.

https://www.biospace.com/article/biospace-fda-decision-tracker-2023-biomarin-celltrans-pfizer-and-opko-health/