Approvals

The US FDA approval of new meningococcal and chikungunya vaccines arrived in time for the 26-28 February meeting of the CDC’s Advisory Committee On Immunization Practices, if political developments allow.

A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include nine new products, one of which is Kostaive (zapomeran), CSL/Arcturus Therapeutics’ self-amplifying mRNA COVID-19 vaccine for individuals 18 years and older.

Mavorixafor, X4 Pharmaceuticals/Norgine's treatment for WHIM syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

GSK’s depemokimab, if approved, could become the first ultra-long-acting drug for treating asthma and chronic rhinosinusitis with nasal polyps. Meanwhile, Sanofi’s teplizumab, which the US approved as the first disease-modifying therapy for type 1 diabetes in 2022, has now been filed for review by the European Medicines Agency.

Top areas for potential 2025 approvals were shaped by R&D focused on increasingly tightly targeted therapies, including the eight new candidates to join the still-burgeoning kinase inhibitor class and RNA interference, as well as many varieties of antibodies.

CBER has at least 14 and CDER another 10 novel biologics among the more than 60 candidates with a user fee goal in 2025.

Among the more than 100 the drug center actions while the FDA's communications have been constrained are approvals of two novel agents, two new indications, and a new combination therapy.

Etuvetidigene autotemcel, Fondazione Telethon’s investigational gene therapy for Wiskott-Aldrich syndrome, is among the latest drugs that have been filed for review by the European Medicines Agency for potential EU marketing approval.

The FDA announcement of the novel, non-opioid drug’s approval highlights public health implications, which probably helped it clear the communications freeze. Recent website updates and forthcoming Federal Register notices suggest the freeze could be thawing slowly.

Only one-third of novel agents with 2025 goal dates come from the traditional oncology, hematology and neuroscience strongholds. Immunodermatology also is cooling, while cardiovascular drugs return to the front burner.

Medicines containing a new active substance (NAS) approved for pan-EU marketing last year were again led by cancer drugs, with treatments for blood disorders following closely behind. Meanwhile, three NAS-containing drugs have been approved so far this year, and 12 other products are expected to be authorized shortly, having recently been recommended for approval by the European Medicine Agency.

Faster approvals and regulatory flexibility are among moves India has made to draw clinical trial investments. As MNCs seek to replace products close to patent expiry with new blockbusters and domestic companies tap unmet needs, how will the R&D landscape shape up in 2025?

The European Medicines Agency’s chief medical officer, Steffen Thirstrup, said the surge in positive opinions adopted by the agency in 2024 for new marketing approvals signals a post-COVID recovery.

Pink Sheet editors consider ex-FDA officials’ advice for the Trump Administration on implementing FDA reforms, comments CDER Director Patrizia Cavazzoni made before her departure from the agency about wanting to release review documents for applications that received complete response letters, and diverging trends between CDER and CBER novel application approvals.

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include two new products, one of which is Gohibic (vilobelimab), InflaRx’s treatment for adult patients with SARS-CoV-2-induced acute respiratory distress syndrome.

The European Medicines Agency wants to work with other regulators as well as industry, scientists and patients to “find solutions for the most pertinent problems in psychedelic research” and improve treatment options for psychiatric disorders more broadly, says the agency.

Novel agents from the biologics center had a median review time of 10.6 months, beating the drugs center's median of 11.8 months thanks to fewer multicycle and more priority reviews.

Review times for all 61 novel agents the US FDA approved in 2024.

Marketing authorization applications for five new advanced therapy medicinal products were filed with the European Medicines Agency in 2024, the highest number in any year since 2020. The Pink Sheet looks at these and two other ATMPs that might get the thumbs up from the European Medicines Agency in 2025.