FDA Action Alert: Biogen/Ionis, Cidara and Incyte

Pictured: FDA Sign/courtesy of Sarah Silbiger/Getty Images

Pictured: FDA Sign/courtesy of Sarah Silbiger/Getty Images

All eyes will be on the FDA’s neuroscience division March 22 as an advisory committee convenes to discuss the fate of Biogen and Ionis’ amyotrophic lateral sclerosis (ALS) hopeful tofersen. Read on for that and more. 

Biogen and Ionis’ Tofersen Faces Adcomm

On March 22, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee will discuss the New Drug Application for an intrathecal injection of tofersen for ALS associated with a mutation in the superoxide dismutase 1 (SOD1) gene.

Tofersen is an antisense oligonucleotide that binds SOD1 mRNA and tags it for destruction, thereby lowering the levels of the faulty protein. Biogen ran the Phase III VALOR study to assess tofersen and in 2021 announced that the candidate fell short of its primary endpoint, inducing no significant improvement in ALS severity.

Still, the biopharma partners went through with their NDA submission, which the FDA first accepted in July 2022. In their submission, the companies pointed to biomarker data: tofersen significantly lowered levels of the SOD1 protein and neurofilament light chain (NfL), which could indicate that the drug is indeed slowing neurodegeneration.

In October 2022, the FDA pushed back tofersen’s target action date by three months, following additional information from Biogen, which the regulator deemed to be a major amendment to the NDA. A verdict on  tofersen is expected by April 25.

The upcoming adcomm meeting will also ripple across the neurodegenerative disease space at large, which in recent years has increasingly been using surrogate biomarkers to measure candidate efficacy.

Moment of Truth for Cidara’s Candida Candidate

On March 22, the FDA is set to decide on rezafungin, Cidara Therapeutics’ investigational drug for the treatment of candidemia. Rezafungin is also being proposed as prophylaxis for invasive candidiasis and other invasive fungal infections in patients undergoing blood and marrow transplantation.

The candida candidate is a novel, once-weekly echinocandin-based antifungal therapeutic. Cidara is supporting its NDA with data from the global Phase III ReSTORE study and the Phase II STRIVE trial, both of which demonstrated that once-weekly rezafungin was non-inferior to caspofungin, the current standard of care in these indications.

The FDA accepted rezafungin’s NDA in September 2022 and had previously granted the drug its Qualified Infectious Disease Product designation, which is reserved for antibacterial and antifungal candidates targeting life-threatening or otherwise serious infections.

Incyte’s Extended-Release Ruxolitinib Awaits Verdict

On March 23, the FDA will decide on Incyte’s extended-release formulation for Jakafi (ruxolitinib). Under this proposed formulation, Jakafi will be given at a once-daily dose.

Incyte came up with this Jakafi formulation as part of its ongoing LIMBER clinical development initiative, which seeks to provide more therapeutic options for patients with myeloproliferative neoplasms.

Data for extended-release ruxolitinib were presented at the 2021 European Hematology Association (EHA) Virtual Congress and showed that the proposed formulation had a bioequivalent pharmacokinetic profile as compared to its immediate-release counterpart. This was true for single and multiple doses of ruxolitinib.

The extended-release formulation was also safe and triggered no new safety signals, according to Incyte.

Correction: An earlier version of this article listed a March 20 PDUFA date for argenx' SC efgartigimod for generalized myasthenia gravis. The FDA pushed this PDUFA back to June 20. 

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