Rozanolixizumab

The FDA approved a new rare disease drug that Johnson & Johnson hopes can compete with established medicines — and some that are yet to be approved — and become a megablockbuster. Regulators on Wednesday gave the thumbs-up to nipocalimab, which will be branded as Imaavy, for patients 12 and up with generalized myasthenia gravis, an autoimmune disease that causes muscle weakness, particularly in the eyes, face, throat and lungs. J&J did not immediately share information about how the drug will be priced. The approval sets up a showdown with Rystiggo and Zilbrysq — both of which are marketed by UCB — as well as argenx’s Vyvgart. Through its $39 billion acquisition of Alexion in 2020, AstraZeneca also has a pair of approved drugs: Soliris and Ultomiris. The FDA’s decision came on time, with the agency expected to approve or reject Imaavy by Tuesday (though J&J did not disclose the PDUFA date). The timing of new drug approvals is under increased scrutiny in light of the widespread cuts at the FDA under Commissioner Marty Makary and HHS Secretary Robert F. Kennedy Jr. Stealth BioTherapeutics on Tuesday reported a delay in the FDA’s decision for its Barth syndrome drug, and a person familiar with the situation said it wasn’t related to safety, efficacy or manufacturing. Myasthenia gravis is a category in which biopharma companies have invested heavily in recent years, and the market is expected to reach between $3 billion and $7 billion by the early 2030s, according to analyst estimates. J&J acquired Imaavy in its $6.5 billion acquisition of Momenta in 2020. Soliris was the first drug to be approved for myasthenia gravis in 2017, but sales have been rising for UCB’s drugs and Vyvgart, all of which have different mechanisms of action than Soliris and Ultomiris and are viewed as the next generation of therapies. In 2024, Rystiggo had $202 million in sales — up from just $19 million in 2023 when it launched — and Zilbrysq reached $72 million in its first year on the market. Vyvgart, meanwhile, has already proved itself a rare disease blockbuster, tallying nearly $2.2 billion in sales in 2024 across its IV and subcutaneous formulations. In addition to myasthenia gravis, Vyvgart also received approval last year in another disease called CIDP, which is when a patient’s immune cells attack the peripheral nervous system. J&J has high hopes for Imaavy, saying it expects the drug can reach more than $5 billion in peak sales across all potential indications, should it continue to win approvals. But to carve out a piece of the market, J&J will have its work cut out for it. Amgen is testing its rare disease drug Uplizna in myasthenia gravis, with a filing expected before the end of June. Immunovant also has a drug in late-stage development, while Biohaven has a program expected to soon start Phase 1 studies in myasthenia gravis. Cartesian Therapeutics is working on a cell therapy program as well.