Pharmaceutical Executive Daily: FDA Grants Ultomiris Accelerated Approval in the U.S.

Welcome to Pharmaceutical Executive Daily, your quick briefing on the top news shaping the pharmaceutical and life sciences industry.

In today’s Pharmaceutical Executive Daily, the FDA grants Ultomiris accelerated approval for adults with primary immunoglobulin A nephropathy based on a 43.4% placebo-adjusted reduction in proteinuria, a pharma funding roundup covers Alcon’s $550 million acquisition of Neurotech Pharmaceuticals and AI Proteins’ collaboration with Astellas worth more than $400 million, and Incubate’s John Stanford argues MFN deals pose the greatest risk to moonshot drug development in cancer, Alzheimer’s, and rare disease.

Alexion, AstraZeneca Rare Disease’s Ultomiris (ravulizumab) has been approved by the FDA under accelerated approval to reduce proteinuria in adults with primary immunoglobulin A nephropathy at risk of disease progression. The approval rests on a pre-specified interim analysis of the Phase III I CAN trial, which showed a 46.6% reduction in urine protein creatinine ratio from baseline at week 34, versus 5.6% on placebo. It marks the fifth indication for the C5 inhibitor, with continued approval contingent on verifying clinical benefit at the trial’s week-106 endpoint.

Two deals closed this week in ophthalmology and protein design. Alcon acquired Neurotech Pharmaceuticals for $550 million plus contingent milestone payments, gaining Encelto, the only FDA-approved cell therapy for macular telangiectasia type 2. Separately, AI Proteins entered a research collaboration and license agreement with Astellas worth more than $400 million in upfront and milestone payments to design novel miniprotein therapeutics against targets selected by Astellas, which holds an exclusive worldwide license to any resulting candidates.

Finally, Incubate executive director John Stanford argues that Most-Favored Nation pricing deals are discouraging companies from pursuing ambitious, high-risk drug development, with cancer and Alzheimer’s already showing measurable drops in innovation investment under the IRA. He contends rare disease is likely the next therapeutic area to feel that pressure, as companies gravitate toward established categories rather than new modalities like mRNA.

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