As the reauthorization deadline nears, CDER and CBER leaders stressed that sunsetting the program would hurt pediatric rare disease development, while endorsing a new Democrat-proposed stick for rare disease research. 

Interactive table from the Pink Sheet breaks down the 40 novel drugs and biologics have user fee goal dates before the new year.

US FDA would need to approve 44 novel agents by year-end to match 2023’s big total, but only 40 candidates are known to have user fee goals in the second half of 2024.

A number of diabetes drugs are also expected to be targeted in the next cycle of the price negotiation program, led by Novo Nordisk’s Ozempic.

Relyvrio/Albrioza, Amylyx’s ill-fated treatment for amyotrophic lateral sclerosis, was approved for marketing by the US Food and Drug Administration but rejected by the European Medicines Agency. The Pink Sheet explores how the two agencies applied the available regulatory flexibilities and the impact of other factors, such as regulatory precedence and patient influence, on decision-making.

Pink Sheet reporter and editors discuss the impact of a US and EU drug approvals analysis that found the US FDA still clears many novel products first, including most new cell and gene therapies and cancer treatments.

Comprehensive table comparing all the novel agents approved by either the US FDA or the European Commission over 2023 through April 2024.

Pink Sheet infographic shows how a large majority of novel drugs make it past the US FDA before receiving approval in the European Union. Lag times ranged from almost five years to less than two weeks.

Editors from the Pink Sheet and Medtech Insight discuss the retirement of Jeff Shuren, PBM executives again appear unscathed after another Capitol Hill appearance, and the growing push to ensure the FDA’s rare pediatric disease priority review voucher program is renewed.

The EMA says the benefits of treatment with Leqembi are not large enough to outweigh its risks. Meanwhile, Alzheimer Europe is hoping that real-world findings from the US-mandated patient registry or from ongoing trials will help persuade the EU regulator to reconsider its position.

Two orphan drugs are among a host of products that the European Medicines Agency’s human medicines committee has OKd for pan-EU approval.

The implementation phase of the European Health Data Space is an opportunity to address “critical aspects” of the new regulation that require clarification, says pharmaceutical industry federation EFPIA.

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered. 

The CDC’s ACIP again punted a decision on whether to make a pneumococcal vaccine available to all adults 50 and older amid concerns over cost and equity tradeoffs, as well as unknowns about the length of vaccine protection. 

Second generation combination products may become more appealing as companies navigate the complexities of the Medicare price negotiation program.

The Oncologic Drugs Advisory Committee urged the FDA not to delay Imfiniz’s perioperative NSCLC indication, while also unanimously pushing for better trial designs in lung cancer and all resectable tumors.

The number of medicines containing a new active substance approved for pan-EU marketing in the first six months of 2024 rose to near-normal levels after an unusually sharp dip in the 2023 period. The approvals were again led by cancer drugs, with therapies for blood disorders a close second, while half of the approvals were treatments for rare diseases.

The UK’s drug regulator said that a trial run by cell therapy firm Celixir “risked seriously jeopardizing the rights, safety and wellbeing of trial participants” after an inspection discovered expired product batches, unauthorized dosing and other serious breaches.

EU member states have agreed to routinely use a flexible process that can facilitate the use of multilingual packaging for prescription-only medicines.

The FDA’s experience with product-specific guidances for generic drugs may impact biosimilar industry decision-making, but the program has improved in recent years.