Product Reviews

The European Medicines Agency has said that Anavex’s Alzheimer’s candidate should not be authorized for use in the EU due to safety and efficacy concerns. Seven other products, including two new cancer drugs, received positive opinions.

The first approval under the Commissioner's National Priority Voucher, a re-introduction of domestic production of an antibiotic, took two months to clear, but the timeline will be more challenging for the three pending CNPV applications with efficacy claims.

Several global products have been granted approval recommendations in Japan, while two Japan-originated drugs received a preliminary nod in their home country after being approved first elsewhere through licensees.

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 Lynkuet, Bayer's treatment for moderate to severe vasomotor symptoms associated with menopause or endocrine therapy for breast cancer.

Saol Therapeutics cannot conduct a new clinical trial of SL1009 in ultra-rare mitochondrial disorder, but aims to answer the FDA's complete response letter with new looks at available data.

Facilities that are not ready for an inspection can lead to delays in the FDA’s site assessment and leave little time to resolve deficiencies ahead of a user fee goal, CBER compliance office Director Melissa Mendoza said.

A cluster of CRLs for rare disease applications based on one trial plus confirmatory evidence may represent a shift away from the regulatory flexibility that had come to characterize ultra-rare drug development

US FDA’s rejection of Biohaven’s troriluzole is a huge disappointment for patients with SCA and the company. But it is also a notable example of how communication strategies are changing now that FDA is making its ‘complete response’ letters public.

Saol Therapeutics cannot conduct a new clinical trial of SL1009 in ultra-rare mitochondrial disorder, but aims to answer the FDA's complete response letter with new looks at available data.

FDA reviewers cited several limitations of the videos, including a lengthy recall period, use of the term “today” in questions, and a lack of standardization in interview conduct.

The Pink Sheet’s Drug Review Profile looks at the clinical development and US FDA review timeline for Stealth's elamipretide for Barth syndrome.

Stealth BioTherapeutics' diligent response to the FDA’s accelerated approval suggestion, its justification for proposed confirmatory trial milestones, and the rarity of Barth syndrome gave the agency confidence to grant approval before the postmarketing study began.

The European Medicines Agency has said that Anavex’s Alzheimer’s candidate should not be authorized for use in the EU due to safety and efficacy concerns. Seven other products, including two new cancer drugs, received positive opinions.

The European Medicines Agency scheduled oral explanation meetings for Sanofi’s MS drug and two other products nearing the end of their EU regulatory review cycle. These meetings typically give companies a final chance to convince the agency their drugs merit approval.

Seven new products, including Anavex’s Alzheimer’s candidate and GSK’s add-on asthma therapy, are up for an opinion from the European Medicines Agency on whether they should be authorized for use in the EU.

Denecimig, Novo Nordisk’s prophylaxis treatment for hemophilia A, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Medical device sponsors can use real-world evidence without submitting identifiable patient-level data under a new guidance that is expected to expand to drugs and biologics.

As differences emerged between FDA staff and senior political leaders over its COVID-19 safety review, CBER Director Vinay Prasad added an old colleague and critic of US COVID-19 policy to the center.

The FDA included storage and handling instructions common in labeling in its approval announcement, suggesting concerns remain about the potential for NDMA to form after products are distributed.

Incentives are “probably the only solution” to encouraging companies to optimize cancer drugs, but this will require funding and systemic changes, the chair of the European Medicines Agency’s oncology working party says.

The tilt toward approval extended beyond patients and advocates to clinicians, professional societies and industry.

At a CDC vaccine advisory committee criticized for “promoting an anti-vaccine agenda,” the new CDER director championed the committee’s plans and suggested the FDA would make more frequent vaccine label updates due to revised safety frameworks.

Because combination vaccines remain in the US childhood vaccine schedule, ACIP's reconsideration of hepatitis B vaccine dose timing could impact uptake of other immunizations, Demetre Daskalakis told the Pink Sheet.

From how he will interact with industry to his staff, to a possible advisory committee revival and dangling accelerated approval crackdowns, Pink Sheet looks at how Rick Pazdur's oncology track record could translate to CDER.