Product Reviews

Two US FDA advisory committees will discuss results from epidemiological studies that attempted to quantify the prevalence, incidence and risk factors for misuse, abuse, overdose and death with opioid analgesics.

US CDC’s Advisory Committee on Immunization Practices voted for harmonized meningococcal vaccine recommendations, while hearing updates about Sanofi’s pending supplemental MedQuadfi indication, AstraZeneca’s FluMist self-administration launch plans, and Lyme disease

Bavarian Nordic's Vimkunya added the US CDC immunization committee's recommendation to its FDA approval, while Valneva's Ixchiq faces a precaution for use in people aged 65 and older.

All RSV vaccines with US FDA approval for high-risk younger adult patients will be incorporated in US CDC’s Advisory Committee for Immunization Practices recommendations. Merck’s infant RSV antibody is on track for a June vote

Plozasiran, Arrowhead Pharmaceuticals' treatment for familial chylomicronemia syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

PharmaMar, which wants to use the Jazz Pharmaceuticals-partnered drug, lurbinectedin, in combination with Roche’s Tecentriq for treating extensive-stage small cell lung cancer, is one of two companies that this month sought an accelerated assessment of their planned EU marketing applications.

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.

The European Medicines Agency has recommended 16 drugs for EU-wide approval, including Vertex’s cystic fibrosis drug Alyftrek and five other orphans.

Plozasiran, Arrowhead Pharmaceuticals' treatment for familial chylomicronemia syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.

Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.

CEO Len Schleifer said during the company’s first quarter earnings call that most of the complete response letters the company has received related to third-party suppliers, not efficacy or safety.

Failing to meet the 29 April user fee date for the Barth syndrome treatment, and CDER’s request for a delay in responding to Vanda’s appeal of a tradipitant complete response letter, suggest a slowdown in review work due to layoffs and other recent changes.

Organizational turmoil has not yet affected reviews of novel agent applications, with Q1 2025 approvals coming in low, but the first quarter share of the entire year is variable.

While CMC glitches linger over a US NDA for Elevar/Hengrui’s novel liver cancer combination following a second complete response letter, the separate issue of underrepresentation of US patients in multiregional trials is looming large after new FDA draft guidance last year.

The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.

Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.

The Pink Sheet drug review profile investigates the long and rigorous process behind the FDA’s recent relaxation of the cardiovascular safety warning for testosterone products.

In an unusual move, Tracy Beth Høeg, a special assistant to FDA Commissioner Martin Makary, is leading the continued negotiations on Novavax’s delayed COVID-19 vaccine approval.

Pink Sheet reporter and editors discuss the most recent senior staff departures at the FDA and their impact on the agency, as well as Commissioner Martin Makary’s plans for a new approval pathway and a combined adverse event database as outlined in an interview with a podcast host.

In discussing FDA’s adverse event monitoring, Makary also seemed to falsely imply the agency did not fully investigate the myocarditis signal with COVID-19 vaccines.

Regulatory authorities from multiple regions are considering extending a pilot project related to collaborative assessments of post-approval chemistry, manufacturing and controls changes, with a focus on the supply of critical medicines.