India Pharma Outlook Team | Saturday, 26 September 2026
The latest regulatory updates point to the FDA backing two oral therapies while it holds the line on early approval.
The agency cleared Mirum’s once-daily pill for a rare bone disorder and AbbVie’s once-daily pill for Parkinson’s disease.
At the same time, Merck and Daiichi Sankyo withdrew their US application for accelerated approval of a lung cancer antibody-drug conjugate after the FDA determined the mid-stage data did not meet requirements.
These decisions together reveal a consistent posture. The FDA enables innovative oral treatments for high-unmet-need conditions yet continues to demand robust evidence before granting accelerated pathways.
Patients gain new convenient options while sponsors receive a clear signal that mid-stage data alone will not secure early green lights.
The FDA approved Mirum Pharmaceuticals’ zilurgisertib, branded Atebrioz, for patients aged 12 years and older with fibrodysplasia ossificans progressiva (FOP). FOP causes muscles, tendons, and ligaments to transform gradually into bone, restricting movement and leading to severe disability. About 300 people in the US and 900 worldwide live with the condition.
Atebrioz is a once-daily oral drug that blocks ALK2, a protein abnormally active in most FOP patients and responsible for extra-skeletal bone formation. The recommended dose is 100 milligrams.
Key points from the approval:
Mirum CEO Chris Peetz described the medicine as a game-changer that can stop the progressive accumulation of bone. Analysts estimate potential peak worldwide sales around USD 150 million.
Also Read: How Can Indian Pharma Strengthen Quality Amid Rising FDA Scrutiny
The FDA also approved AbbVie’s tavapadon, to be sold as Juvmo, as a once-daily pill for Parkinson’s disease in adults. Parkinson’s is a long-term brain condition that gradually damages movement control and often causes tremors, stiffness, and balance issues. More than 11 million people live with the disease worldwide.
Juvmo works by mimicking dopamine, the brain chemical that decreases in people with Parkinson’s. The approval drew on data from AbbVie’s late-stage trial program that showed improvement in symptoms. Common side effects included nausea, dizziness, and headache.
AbbVie gained the asset through its USD 8.7 billion acquisition of Cerevel Therapeutics. The company has not commented on launch timing or pricing. Analysts previously expected a 2026 launch with gradual uptake pending Medicare coverage discussions.
Merck and Daiichi Sankyo withdrew their US application seeking accelerated approval for ifinatamab deruxtecan. The companies took this step after discussions with the FDA. The agency determined that data from a mid-stage clinical study did not satisfy the requirements needed to support an early green light.
The therapy targets adults with extensive-stage small cell lung cancer whose disease had worsened after standard chemotherapy. It belongs to the antibody-drug conjugate class that delivers cytotoxic payloads to tumor cells.
This marks the second withdrawal under the companies’ collaboration worth up to USD 22 billion. Last year they pulled the application for another lung cancer candidate after it failed to extend survival in a late-stage study.
Despite the setback, the partners will continue evaluating ifinatamab deruxtecan. Patient enrollment is nearly complete for a larger late-stage trial comparing the therapy against standard chemotherapy options. They plan to use those results to seek full approvals from the FDA and other global regulators. The drug is also in late-stage trials for advanced prostate and esophageal cancers.
The FDA approved two convenient oral therapies that address significant unmet needs, one for an ultra-rare progressive bone disease and one for a widespread neurological condition. At the same time, the agency required stronger evidence before granting accelerated approval to a mid-stage cancer candidate.
These actions show the regulator enabling innovation for patients who need better options while maintaining rigorous standards for early approval pathways. Sponsors now have a single-day illustration of both the opportunity and the evidentiary bar.
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