August’s 6 Most Important Drug Updates
We tracked 31 pharma news catalysts in August across approvals, new indication approvals, positive Phase 3 results and NDA updates. Here are the six that matter most — Lipfendra, AMT-130, cemdisiran, ivonescimab, mezigdomide and Gazyva.
See more detail on the updates below.
Lipfendra — Dyslipidemia — Merck
Merck’s Lipfendra is the first oral PCSK9 inhibitor approved by the FDA, with Phase 3 LDL-C reductions of 56% and 59% versus placebo. More than 20 contacts sit across the atherosclerosis franchise. Projected peak sales: $5.0B.
AMT-130 — Huntington’s Disease — uniQure
uniQure will file for accelerated approval within months after the FDA agreed to accept three-year Phase 1/2 data, where the higher dose slowed disease progression by 75% versus natural history. Projected peak sales: $2.0B.
Cemdisiran — Myasthenia Gravis — Regeneron
Regeneron’s NDA has been accepted under Priority Review, with a November target date. Its rare disease franchise is a strong target right now, with Otarmeni approved and garetosmab awaiting a decision. Projected peak sales: $1.9B.
Ivonescimab — Lung Cancer — Summit
Summit’s updated analysis showed a 24% reduction in risk of death in previously treated EGFR-mutated NSCLC, though short of statistical significance ahead of a November 14 decision. The commercial team is established and growing. Projected peak sales: $1.7B.
Mezigdomide — Multiple Myeloma — BMS
The FDA accepted BMS’s NDA in relapsed or refractory multiple myeloma, after Phase 3 data showed a 52% reduction in risk of progression or death. Its CELMoD franchise is one to watch, with iberdomide also awaiting approval. Projected peak sales: $1.5B.
Gazyva — pMN — Roche
Roche’s anti-CD20 antibody keeps moving out of oncology into immune-driven kidney disease. Approved in lupus nephritis last October, it is now under Priority Review in primary membranous nephropathy, with a decision due November 2026.
Full update in our Insights Hub, a free resource for pharma pipeline insights.