In
August, the global pharmaceutical industry witnessed several regulatory
upheavals and policy shifts in the US. The month began with the US President
Donald Trump announcing that planned tariffs on drug imports into the US may reach 250 percent within 12 to 18 months.In the last days of July, Vinay Prasad, the head of the US Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER) had stepped down (reportedly under pressure). On August 12, Prasad was back at CBER as its head. Then,
around the month end, the White House fired Centers For Disease Control and
Prevention (CDC) director Susan Monarez after a clash with
Health and Human Services (HHS) Secretary Robert F. Kennedy Jr. Four senior CDC
officials resigned soon after.Despite this chaos, the pharma indices showed resilience and climbed upwards. The Nasdaq Biotechnology Index (NBI) rose 5.22 percent from 4,445.39 to 4,677.63. The SPDR S&P Biotech ETF (XBI) gained 5.92 percent from 84.83 to 89.85. And the S&P Biotechnology Select Industry Index (SPSIBI) climbed 4.9 percent from 6,671.56 to 6,998.36. Access the Pipeline Prospector Dashboard for August 2025 Newsmakers (Free Excel) Novo’s Wegovy gets approved for MASH; Precigen’s Papzimeos okayed for rare respiratory conditionThe
FDA granted accelerated approval to Novo Nordisk’s Wegovy (semaglutide) for treating metabolic-associated steatohepatitis, or MASH — a progressive liver disease linked to obesity. MASH causes inflammation and scarring of the liver. This approval makes the blockbuster obesity drug the first glucagon-like peptide-1 (GLP-1) receptor agonist approved for this condition. The
US regulator also granted an accelerated approval Hernexeos (zongertinib), a new drug from Boehringer Ingelheim, for adults with advanced
non-small cell lung cancer (NSCLC) with specific HER2 mutations who have
undergone prior treatment. In studies, the drug helped 75 percent of patients
who had chemotherapy see their cancer shrink or disappear completely.Insmed (stock up 25 percent in
August) secured FDA approval for its med Brinsupri (brensocatib). This is the first treatment for non-cystic fibrosis
bronchiectasis, a chronic lung disease that leads to persistent cough,
recurring infections, and damage to the airways.Maryland-based
biotech Precigen bagged FDA approval for
Papzimeos (zopapogene imadenovec-drba), making it the first treatment for recurrent respiratory
papillomatosis. This is a rare condition that causes benign growths in the airway and
voice box. Patients often require multiple surgeries each year to remove these
growths, and until now there was no approved drug for this condition. Precigen
saw its stock shoot up 182 percent during the month. Access the Pipeline Prospector Dashboard for August 2025 Newsmakers (Free Excel) Ionis’ Dawnzera becomes first RNA-targeting therapy for angioedema; Tonix’s fibromyalgia med okayedIonis Pharmaceuticals gained FDA approval for
Dawnzera (donidalorsen), the first RNA-targeting preventive therapy for
hereditary angioedema. This rare genetic disorder causes sudden and
unpredictable swelling that can be life-threatening if it affects the airway.New
Jersey-based biotech Tonix Pharmaceuticals received approval for Tonmya (cyclobenzaprine), the first new fibromyalgia
treatment in over 15 years. Fibromyalgia is a chronic disorder that causes
widespread pain, fatigue, sleep problems, and memory issues.Teva Pharmaceuticals won an expanded indication for Ajovy (fremanezumab) to prevent migraines in children between six and 17 years old.Jazz Pharmaceuticals reported a mix of clinical
and regulatory developments. Its drug Modeyso (dordaviprone) received accelerated approval for diffuse midline glioma,
a rare and aggressive brain tumor that occurs in both children and adults. Jazz
also strengthened its pipeline by licensing SAN2355, an epilepsy drug candidate, from Danish
biotech Saniona. Access the Pipeline Prospector Dashboard for August 2025 Newsmakers (Free Excel) Novartis’ Sjögren’s disease med posts trial win; IO Biotech’s melanoma vaccine faces phase 3 setbackNovartis reported success for its investigational
monoclonal antibody ianalumab in a phase 3 study in patients with Sjögren’s disease. This is a chronic autoimmune condition in which the body’s immune system attacks its own moisture-producing glands, leading to dry eyes, dry mouth, and other complications. Similarly, Regeneron announced positive results for cemdisiran in generalized myasthenia
gravis, a rare condition that causes muscle weakness.In
negative news from trials, IO Biotech announced results from a late-stage trial of its experimental melanoma
vaccine in combination with Merck’s Keytruda (pembrolizumab). Melanoma is a serious form of skin cancer. The vaccine’s benefits were not statistically significant. This setback dragged IO Biotech’s stock down by 28 percent over the month. The company still plans to file for FDA approval.Allogene reported a patient death in a phase 2 trial on its CAR-T cancer therapy — cemacabtagene ansegedleucel. And Agios Pharmaceuticals disclosed that four patients taking its anemia drug Pyrukynd (mitapivat) had died, according to FDA’s Adverse Event Reporting System (FAERS) database.Valneva faced a different kind of setback — FDA suspended the use of its chikungunya vaccine Ixchiq after 21 hospitalizations and three patient deaths, including one linked to encephalitis, a type of brain inflammation. With Ixchiq off the market, Bavarian Nordic’s Vimkunya is now the only chikungunya vaccine available in the US. Access the Pipeline Prospector Dashboard for August 2025 Newsmakers (Free Excel)Merck KGaA-Skyhawk in US$ 2 bn neurology deal; AbbVie acquires Gilgamesh’s psychedelic drugMerck KGaA struck a strategic research deal worth more than US$ 2 billion with Massachusetts-based Skyhawk Therapeutics to develop RNA-targeting small molecules for neurological conditions. The deal,
valued at over US$2 billion, includes upfront and
milestone payments as well as tiered royalties on future product sales. Under
the agreement, Skyhawk will lead discovery and preclinical work, while
Merck KGaA will assume development and commercialization.The
month saw several sub-US$ 2 billion deals. Bayer signed a US$ 1.3 billion agreement with
California-based Kumquat Biosciences to develop a cancer drug targeting KRAS
mutations. These mutations are among the most common in cancer and occur in roughly 25 percent of cases. Eli Lilly entered a US$ 1.3 billion partnership with
Boston-based Superluminal Medicines, an artificial intelligence firm. The collaboration aims
to identify small-molecule drugs for obesity and metabolic diseases.Jazz Pharmaceuticals also licensed an epilepsy therapy from
Denmark-based Saniona in a deal that could reach US$ 1 billion in value. And AbbVie acquired rights
to Gilgamesh’s experimental psychedelic
drug for depression for up to US$ 1.2 billion.Moreover,
there were reports of Novartis planning to acquire San
Diego-based Avidity Biosciences in early August. Avidity is valued at about US$ 5.8 billion. Access the Pipeline Prospector Dashboard for August 2025 Newsmakers (Free Excel) Our viewWhile it may seem like it’s business as usual in the global biopharma industry, the recent slowdown in big-ticket M&A deals suggests undercurrents of caution. Innovation and strong pipelines are keeping the momentum alive, but escalating geopolitical tensions, drastic cuts in federal funding in the US and, a rise in protectionism the world over are forcing companies to rethink their risk appetite. This conservative shift, though understandable, also signals that innovation and global collaboration may stand to suffer. Access the Pipeline Prospector Dashboard for August 2025 Newsmakers (Free Excel)
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