The FDA is recruiting to fill vacancies on its advisory committees, informally known as adcomms. While these committees have met less frequently during the Trump administration, more meetings have been scheduled since former Commissioner Marty Makary left the agency in May.
Patients obtaining drugs through social media, medical spas, or telehealth platforms are not the same as patients obtaining drugs through more traditional illicit channels because they often believe what they are receiving is a clinically validated product. This means they may present with unexplained symptoms without realizing their online purchase is even relevant to the conversation
We have the opportunity to design systems that give people greater capacity for the work where human presence matters most: listening, understanding, reassuring, deciding, and caring.
Mirum Pharmaceuticals’ Atebrioz is the third FDA-approved drug for fibrodysplasia ossificans progressiva (FOP), but it brings advantages over the two other products for this rare disease that leads to bone growth in soft tissue. Mirum acquired Atebrioz from Incyte earlier this year.
As a country, we have developed treatments capable of improving the quality of life and preventing long-term damage for children with rheumatic conditions, yet many children cannot obtain them when they need them most. This needs to change.
Ultragenyx Pharmaceutical’s Fayuvi is now the first FDA-approved therapy for the underlying cause of Sanfilippo syndrome type A, an ultra-rare inherited neurological disorder. It’s the second approved gene therapy for Ultragenyx, which specializes in rare disease medicines.
Scholar Rock’s Isembyld isn’t the only FDA-approved therapy for spinal muscular atrophy, but it is the first one that directly targets muscle tissue. Isembyld is the first commercial product for Scholar Rock, which is also evaluating the antibody in other rare muscle disorders.
What if health plans could identify member decline before an avoidable hospitalization occurs? Real-time clinical visibility into long-stay SNF members uncovers risk earlier and drives better outcomes.
Ionis Pharmaceuticals’ zilganersen, brand name Zanvastro, is the first disease-modifying therapy approved for the neurological disorder Alexander disease. While Ionis has experience developing medicines for rare neuroscience indications, Zanvastro will be the first neurology product the company brings to the market without a commercialization partner.
Roivant Sciences subsidiary Priovant Therapeutics received FDA approval for Lisraya as a treatment for the rare inflammatory disorder dermatomyositis. Licensed from Pfizer, this once-daily pill designed to block JAK1 and TYK2 proteins is also in clinical development for three additional rare immunological disorders, each with blockbuster potential.
FDA approval of Revolution Medicines’ daraxonrasib, brand name Rasonque, makes the molecule the first RAS inhibitor approved for treating pancreatic adenocarcinoma, the most common type of pancreatic cancer. The speedy regulatory nod specifically covers advanced cases of this type of cancer, which has had limited treatment options.
Johnson & Johnson antibody drug Imaavy expanded its label to include the treatment of warm autoimmune hemolytic anemia (wAIHA). Projected to become a blockbuster seller across multiple indications, Imaavy was first approved last year for treating generalized myasthenia gravis.
Healthcare decision-makers, especially at pharmaceutical companies, must prioritize patient input. Data collected from social media listening is a significant step in the right direction.
Ultragenyx Pharmaceutical’s Genglycos received accelerated FDA approval for glycogen storage disease type Ia, an inherited enzyme deficiency. It’s also the first approved gene therapy for Ultragenyx, which specializes in rare disease treatments.
Why the flood of consumer wellness devices demands clear boundaries between lifestyle improvement and clinical diagnostics
Bristol Myers Squibb’s Zenbexus is the first FDA-approved drug in a new class of cancer drugs called CELMoDs. The pharma company is positioning this molecule as a successor to its legacy products for multiple myeloma.
CMS unveiled new details of its new RAPID pathway, which is designed to close the gap between FDA approval and Medicare coverage for breakthrough medical devices. Industry reaction has been largely positive, as medical device companies often sit in “coverage purgatory” for years.
Replimune went from an FDA rejection to a resubmission, a positive advisory committee vote, and finally an accelerated regulatory approval for advanced melanoma — all in less than four months. The FDA nod for Replimune’s oncolytic viral therapy, Tudriqev, positions it as a more convenient alternative to an Iovance Biotherapeutics cell therapy that had been the only treatment in this setting.