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The FDA in October 2025 granted bitopertin its Commissioner’s National Priority Voucher but after a shortened review time has decided the data did not support regulatory approval for treating erythropoietic protoporphyria.
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The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is part of a larger communications crisis unfolding at the agency over the past nine months that has also ensnarled Sarepta, Capricor, uniQure and many more.
The rare disease drugmaker is facing potential competitors for achondroplasia drug Voxzogo. Is a big M&A deal with two approved assets enough to maintain investor interest?
The FDA issued a rare Refusal-to-File letter to Moderna over its mRNA-based influenza vaccine application, in an unusual move that sent the biotech’s shares tumbling.
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Novo Nordisk and Eli Lilly have been battling head-to-head in an exploding obesity market. They should never have been compared apples to apples.
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Despite Ironwood Pharmaceuticals’ apraglutide getting positive late-stage study results in treating short bowel syndrome with intestinal failure, the stock plummeted nearly 40% on Thursday.
The annual observance highlights how far the industry has come and serves as a wake-up call for how far it must still go to meet the needs of patients with rare diseases.
Antibody-drug conjugates are a breakthrough in targeted cancer therapy and will continue to attract big pharma investment over the next few years, finds a new report from market intelligence firm Evaluate.
The FDA and CDC warned Thursday of a small increased risk of developing Guillain-Barré syndrome in older adults after immunization with respiratory syncytial virus vaccines from GSK and Pfizer.
Retaining the full rights to Trodelvy, Gilead now has access to $210 million in financing from venture capital firm Abingworth to help push the antibody-drug conjugate into non-small cell lung cancer studies.
Despite the approval of two novel therapies for this uniformly fatal neurodegenerative disease, experts say regulatory standards and expectations are still evolving.
Denmark-based startup Orbis Medicines has launched with €26 million in seed funding in its drive to search for macrocycle drugs, with a focus on high-value oral alternatives to blockbuster biologics.
The FDA will kick off March with three target action dates, including one for an insomnia treatment and another for a multiple sclerosis therapy.
Eli Lilly is planning to launch its blockbuster diabetes drug Mounjaro next year in India, the world’s most populous nation, ahead of its main competitor Novo Nordisk which is eyeing an Indian launch of Wegovy in 2026.
Sarepta Therapeutics on Wednesday called the launch of the gene therapy for Duchenne muscular dystrophy “exceptional” but the company does not expect to see significant growth in the first half of 2024 due to its currently limited patient pool.