Approval Standards

Exploring How Everyday Industrial Compounds Enter Our Systems, Their Health Implications, and the Ongoing Quest for Safer Alternatives .

With the deadline for comments on the US FDA’s draft guidance less than a month away, the Pink Sheet offers an infographic outlining what the agency is expecting in diversity action plans and when that might be implemented.

The agency also should explain the potential consequences for sponsors if enrollment goals are not met, stakeholders say in comments on the DAP draft guidance. Some commenters urged the FDA to apply waiver criteria flexibly, especially for rare diseases.

The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.

ES: A second pivotal study may be enough to get Iterum’s antibiotic through its second FDA review, but data gaps raise concerns about inappropriate prescribing contributing to antimicrobial resistance and could limit marketing differentiation.

Even as US FDA expressed concern about the perioperative trial design, AstraZeneca’s ability to snag NSCLC indication without demonstrating the benefits of both the neoadjuvant and adjuvant phases of therapy highlights slow pace of agency policy shifts.

The agency’s final guidance retains a draft recommendation for a randomized, parallel dose-response trial when comparing multiple dosages, but is more forceful in wanting sponsors to talk to the FDA early if they want advice tailored to their development programs.

AstraZeneca’s Imfinzi lung cancer advisory panel, which offered insight into why sponsors do not always follow agency advice, likely is a preview of the hurdle facing Bristol Myers Squibb’s Opdivo application for the same indication. 

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

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.

The Oncologic Drugs Advisory Committee will vote on a product agnostic question related to perioperative trial designs during a 25 July meeting after discussing whether AstraZeneca needs an additional trial for its Imfinzi perioperative regimen in non-small cell lung cancer.

Senior leadership in the FDA’s drugs, biologics and devices centers want reviewers to feel more comfortable taking risks in product approvals, but may need more concrete examples of regulatory flexibility's success to convince them.

CDER real world evidence policy head John Concato said the FDA could approve a new labeled use based only on real-world evidence, but made clear the approach is challenging. 

Novel agents in immuno-oncology headline the drug center’s upcoming goal dates, while gene therapies make up most of the biologic center’s workload.

So far this year, when the agency has taken action on NMEs that went before advisory committees, the FDA decision has matched the committee vote.

Half of the FDA’s upcoming 2024 user fee goals target rare diseases, but highly prevalent conditions like uncomplicated urinary tract infections and atopic dermatitis are also up for agency action in the second half of the year.

Firms can be more forceful in disputes with FDA now that Supreme Court has eliminated Chevron deference. Questions around whether an evidentiary standard has been met may be ripe for challenge, legal experts said, but they also caution that sponsors will face more uncertainty.

The European Medicines Agency says that in 2022, the average time taken up by clock-stops exceeded the average time of drug assessment.

Clene leadership spoke with the Pink Sheet about its push for accelerated approval for its ALS nanoparticle treatment. A new nudge from Congress asking the US FDA to consider survival data in accelerated approval could help.