Navigating the FDA Regulatory Maze: Challenges Faced by These Resilient Companies

This week, the FDA served up a harsh reminder to pharmaceutical companies about the challenging path to drug approvals. ARS Pharmaceuticals, despite gaining support from the Pulmonary-Allergy Drugs Advisory Committee earlier in May, faced a rejection from the FDA for its neffy nasal spray designed to combat allergic reactions. This episode underscored the FDA’s discretion, as it doesn’t always follow the advice of its advisory committees. ARS, undeterred, plans to appeal this decision by filing a Formal Dispute Resolution Request.

On another front, Intarcia Therapeutics saw its ITCA 650 implantable drug-device combination for treating type 2 diabetes unanimously rejected by the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee, marking the latest chapter in ITCA 650’s turbulent regulatory journey, which already included two Complete Response Letters (CRLs).

Taysha faced a tough call this week as it dropped its lead gene therapy candidate for giant axonal neuropathy due to the FDA’s insistence on a randomized, double-blind, placebo-controlled trial. Adding to the disappointment, Taysha also revealed that Astellas was no longer pursuing an exclusive license for the therapy.

BeiGene managed to navigate regulatory hurdles, with Novartis returning the former’s anti-PD-1 antibody. This move granted BeiGene global rights to develop, manufacture, and commercialize tislelizumab. Additionally, the FDA accepted BeiGene’s Biologics License Application for tislelizumab as a first-line treatment for esophageal squamous cell carcinoma.

While some companies rebounded from regulatory setbacks, like Alvotech, whose Biologics License Application for a Humira biosimilar was accepted after facing two CRLs, others, like Mesoblast, gained clarity on advancing their cell therapy for acute graft-versus-host disease following a Type A meeting with the FDA.

In the world of kidney disease, Travere Therapeutics faced a setback when its confirmatory Phase III study for sparsentan narrowly missed a crucial endpoint. Despite this, the company plans to meet with regulators and aims to submit a supplemental New Drug Application for full approval in the first half of 2024.

Amidst these challenges, progress shone through in late-stage cancer trials. Merck, Seagen, and Astellas celebrated a significant milestone as their Phase III trial met dual primary endpoints in the quest to treat locally advanced and metastatic urothelial cancer. Simultaneously, AstraZeneca and Daiichi Sankyo reported positive outcomes for their antibody-drug conjugate in a late-stage breast cancer study, setting the stage for potential competition with Gilead’s Trodelvy.

Finally, in honor of World Cancer Research Day on September 24, BioSpace delved deep into 10 promising therapeutic candidates poised to revolutionize the landscape of cancer treatment, offering hope to those affected by lung, breast, colorectal, and other cancers.

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