Pink Sheet Perspectives

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.

Restrictions on commonly used chemicals, increased reporting requirements and enhanced environmental risk assessments are just some of the new EU sustainability and environmental rules pharmaceutical companies are potentially facing. While some measures are multi-sectoral, others are pharma specific. The Pink Sheet takes a look at some of the developments in 2024 and expectations for 2025 and beyond.

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.

The inspector general said the council should be more involved in specific approval decisions when there is internal disagreement, but FDA said that would preclude some individuals from adjudicating a subsequent withdrawal proceeding.

Two recent accelerated approval guidances describe the FDA’s policy expectations for trial timeliness, but recognize the need for case-specific determinations. Regulatory actions in 2024 show the FDA is willing to delay application filing or approval over confirmatory trial status.

While Japan looks set to further improve policies to support ventures and ease market entry this year, these will contrast with ongoing reimbursement price controls.

Prospects are promising for many of the 16 novel candidates that received complete response letters in 2024 after half of the 2023 CRL class was approved the following year.

Too few marketing authorization applications are submitted to the European Medicines Agency on time, leading to disruption and resourcing difficulties. The Pink Sheet examines initiatives taken in the recent past to address the issue and what companies need to be aware of to ensure the evaluation process is as efficient as possible.

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?

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 two-time FDA commissioner resigned on 20 January as part of the administration change. Califf is credited with righting an agency demoralized from the COVID-19 pandemic, but worries about its future are high.

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.

The European Commission's 100-day countdown to deliver a Critical Medicines Act, aimed at improving the EU's resilience to supply chain disruptions and price volatility, began last December. Meanwhile, negotiations over the proposed package that will reform the over 20-year-old EU pharmaceutical legislation are now in the hands of the Council of the EU. The Pink Sheet examines what to expect for these key pieces of legislation this year.

Now that one of the most controversial pharma M&As of 2024 has closed, the Pink Sheet looks at whether overtures to concerned customers and extended prenotification discussions helped Novo and Catalent seal the deal.

Anti-PBM sentiment continues to rise, but industry observers still question whether the reforms being discussed will be effective.

Regenerative medicines using expedited review pathways dominate novel approvals at the Center for Biologics Evaluation and Research, while the Center for Drug Evaluation’s higher volume comes with lower first-cycle approval rates and more standard reviews.

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.