OptumRx profiles four therapies that are expecting FDA decisions in the next few months.
Two specialty medications to treat rare diseases, along with novel treatments for myeloma and plaque psoriasis, are highlighted in OptumRx’s new Drug Pipeline Insights report. The top four medications —teclistamab, deucravacitinib, betibeglogene autotemcel, and elivaldogene autotemcel— are expected to receive FDA approval in the third quarter.
• Deucravacitinib (brand name to be determined) is a novel oral drug being evaluated to treat adults with moderate-to-severe plaque psoriasis developed by Bristol Myers Squibb. Plaque psoriasis affects about 6 million people in the United States. Deucravacitinib is a novel tyrosine kinase 2 (TYK2) inhibitor that regulates overproduction of immune inflammatory compounds in immune-mediated diseases such as psoriasis, OptumRx said.
While deucravacitinib would provide a first-in-class oral treatment for a condition with limited oral options, it is still entering a crowded marketplace, according to OptumRx.
Injectable biologics are commonly used with high response rates in patients with moderate-to-severe plaque psoriasis. Immunosuppressants such as methotrexate and cyclosporine have historically been used, but their place in therapy is limited because of safety concerns, according to the PBM.
Celgene’s Otezla (apremilast), an oral phosphodiesterase 4 (PDE4) inhibitor, has been available since 2014 and has a safer adverse event profile versus historical oral treatments but its use has been limited because injectable biologics are more effective, OptumRx said. In trials, deucravacitinib demonstrated superior efficacy versus Otezla and was associated with numerically lower rates of discontinuations due to adverse events.
“Deucravacitinib is indeed entering a crowded marketplace with multiple treatments already available,” Bill Dreitlein, Pharm.D., senior director, pipeline and drug surveillance at OptumRx, told Formulary Watch. “However, psoriasis is an $11 billion marketplace and deucravacitinib will aim to carve out a slice of that as a new oral option.”
An FDA decision is expected Aug. 19, 2022.
•Teclistamab (brand name to be determined), administered subcutaneously, is a novel drug to treat relapsed or refractory multiple myeloma developed by Janssen. Teclistamab is a bispecific antibody that targets both B-cell maturation antigen (BCMA) and CD3, a T-cell receptor.
It enters a competitive field, with three similar therapies that target BCMA for relapsed or refractory multiple myeloma, OptumRx said. Existing medications include:GSK’s Blenrep(belantamab mafodotin), which is administered intravenously; along with CAR-T cell therapies Carvykti (ciltacabtagene autoleucel) from Janssen Biotech and Abecma (idecabtagene vicleucel) from Bristol Myers Squibb.
Indirectly, teclistamab appears more effective than Blenrep, but less effective versus the CAR-T cell therapies, according to OptumRx. “However, it appears that the CAR-T cell therapies see higher rates of CRS and neurotoxicity than teclistamab,” the PBM said.
A wholesale acquisition cost for teclistamab has not been announced, according to OptumRx.
An FDA decision is expected Sept.10, 2022.
• Betibeglogene autotemcel, with a brand name of Zynteglo, is a gene therapy for transfusion-dependent beta-thalassemia developed by Bluebird Bio. If approved, Zynteglo could be a cure for the rare disease through a one-time intravenous infusion, according to OptumRx.
Beta-thalassemia is caused by genetic mutations in cells that carry oxygen throughout the body, which may produce classic signs of anemia and may be life-threatening if left untreated, according to OptumRx.
Current treatment for severe beta-thalassemia is mainly regular, life-long red blood cell transfusions. Patients also need iron chelation therapy to address excess levels of iron in the body due to the repeated blood transfusions.
Stem cell transplantation from a matched donor, ideally a sibling, is currently the only possible cure. However, stem cell transplants carry their own risks, and fewer than 25% of patients have access to a suitable match, Dreitlein said.
The only other FDA-approved therapy for treatment transfusion dependent beta-thalassemia is Reblozyl (luspatercept) developed by Bristol Myers Squibb and Acceleron, which only treats the anemia resulting from the disease and requires an injection every three weeks.
“If approved, Zynteglo would be a one-time treatment and the first gene therapy for a blood disorder,” Dreitlein said. As a result, it could “break the ice” for other treatments in the future for other blood disorders such as sickle cell disease, he added.
Of the estimated 3,000 patients with beta-thalassemia in the United States, about half of them are dependent on blood transfusions. “The target population for this treatment is extremely small. Bluebird bio estimates about 1,500 patients with beta-thalassemia in the United States would be eligible,” OptumRx said.
Analysts forecast a $2.1 million cost for a one-time dose of Zynteglo, according to OptumRx.
An FDA decision is expected Aug. 19, 2022
• Elivaldogene autotemcel, with the brand name Skysona, is a gene therapy to treat cerebral adrenoleukodystrophy (CALD) in young boys developed by Bluebird Bio. Patients with CALD lack a protein (ALDP) needed to break down fatty acids, which causes toxic acids to build up in the brain and leads to disability and an early death. Most patients with CALD will die within a decade of diagnosis if they are not treated with bone marrow (stem cell) transplantation.
There are around 40 patients with CALD in the United States annually, according to OptumRx.
“If approved, Skysona would be a one-time treatment that may stabilize disease progression and preserve neurological function in patients with early CALD,” OptumRx said.
While Bluebird bio has not announced a price for Skysona, the wholesale acquisition cost for Zolgensma (onasemnogene abeparvovec), a one-time gene therapy for spinal muscular atrophy, is $2.125 million, according to Optum Rx.
An FDA decision is expected Sept. 16, 2022.