White House sacks CDC director; FDA okays Ionis’ Dawnzera, first RNA-based preventive for hereditary angioedema

By PharmaCompass

2025-08-28

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The Trump administration has fired US Centers for Disease Control and Prevention (CDC) Director Susan Monarez, post a policy disagreement with Health and Human Services (HHS) Secretary Robert F. Kennedy Jr (RFK Jr). Four other senior CDC officials have also resigned over RFK Jr’s vaccine policy.

The US Food and Drug Administration (FDA) has approved Ionis Pharmaceuticals’ Dawnzera (donidalorsen) for treating hereditary angioedema (HAE), a rare genetic disorder. This is the first RNA-targeting therapy cleared as a preventive treatment for HAE.

FDA has suspended the use of Valneva’s chikungunya vaccine Ixchiq in the US, citing 21 hospitalizations and three deaths, including one from vaccine-linked encephalitis (inflammation of the brain).

FDA has issued a Form 483 to Novo Nordisk’s Indiana plant, citing serious quality lapses and contamination risks.

AbbVie has acquired Gilgamesh’s experimental psychedelic drug bretisilocin for US$ 1.2 billion. The drug is being tested for major depressive disorder. The drugmaker’s rheumatoid arthritis med Rinvoq (upadacitinib) has delivered strong results in two phase 3 trials for alopecia areata, a serious autoimmune hair-loss condition.

Meanwhile, Eli Lilly’s oral GLP-1 pill — orforglipron — helped type 2 diabetes patients lose around 10.5 percent of body weight in late-stage studies.

In news from other clinical trials, Akeso and Summit’s ivonescimab extended survival in EGFR-mutated non-small cell lung cancer (NSCLC). And Regeneron’s cemdisiran improved muscle function in patients with generalized myasthenia gravis, a rare disease that weakens muscles.

Trump admin sacks CDC chief within weeks of hiring her; four officials resign

The White House has fired CDC Director Susan Monarez within a month of her appointment. Monarez refused to step down from her post after a policy disagreement with HHS Secretary RFK Jr. This prompted the Trump administration to fire Monarez as the head of America’s public health agency.

According to the White House spokesman Kush Desai, Monarez was not “aligned with the President’s agenda of Making America Healthy Again”.

The move has triggered resignations from at least four senior CDC officials, including the agency’s chief medical officer and leaders of immunization and emerging disease centers. The shakeup comes amid concerns over the agency’s approach to immunizations under RFK Jr, a known vaccine skeptic.

FDA okays Ionis’ Dawnzera, first RNA-based preventive for hereditary angioedema

FDA has approved Ionis Pharmaceuticals’ Dawnzera (donidalorsen) for patients 12 and older with hereditary angioedema (HAE), a rare genetic disorder that causes swelling. This is the first RNA-targeting therapy cleared as a preventive treatment for HAE. The therapy, given as a self-injection every four or eight weeks, carries precautions for allergic reactions. Dawnzera will be launched in the coming days with a list price of US$ 57,462 per dose.

Approves updated Covid-19 jabs for high-risk groups: FDA has approved updated Covid-19 vaccines from Moderna, Pfizer and Novavax , but for a limited group of adults (aged 65 or more) and children with underlying health conditions that put them at higher risk. Vaccines are no longer automatically authorized for healthy younger children, as the FDA has rescinded the broader emergency use approvals.

FDA suspends use of Valneva’s chikungunya vaccine after deaths of three patients

FDA has suspended the use of Valneva’s chikungunya vaccine Ixchiq in the US, citing 21 hospitalizations and three deaths, including one from vaccine-linked encephalitis (inflammation of the brain). Ixchiq had generated €7.5 million (US$ 8.8 million) in first-half 2025 sales, about eight percent of Valneva’s revenue. With Ixchiq grounded, Bavarian Nordic’s FDA-approved Vimkunya is now the only chikungunya vaccine available in the US.

AbbVie buys Gilgamesh’s depression drug for US$ 1.2 billion

AbbVie will pay up to US$ 1.2 billion to acquire Gilgamesh’s experimental depression drug bretisilocin. The drug is being tested for major depressive disorder. Instead of buying the biotech outright, AbbVie is taking over the program while Gilgamesh spins out its other projects into a new company. Bretisilocin is a next-generation psychedelic that has shown to exert a shorter duration of psychoactive experience, while retaining an extended therapeutic benefit.

Meanwhile, AbbVie’s rheumatoid arthritis drug Rinvoq (upadacitinib) has delivered strong results in two phase 3 trials for alopecia areata, a serious autoimmune hair-loss condition. AbbVie now plans to file for regulatory approval, potentially making Rinvoq a new rival to Eli Lilly’s Olumiant, Pfizer’s Litfulo (ritlecitinib), and Sun Pharma’s Leqselvi (deuruxolitinib). The drug is already a blockbuster, generating US$ 5.9 billion in 2024, up nearly 48 percent year-on-year. It is approved to treat several inflammatory diseases.

J&J to invest US$ 2 bn in NC facility: Johnson & Johnson is investing US$ 2 billion over the next 10 years in order to increase its presence in North Carolina with an over 160,000 square foot dedicated manufacturing facility at the Fujifilm site in Holly Springs. This investment will create 120 new jobs, and also help in averting the import tariffs that may become applicable in future.

FDA issues Form 483 to Novo’s Indiana plant; cites cat hair, contamination risks

Novo Nordisk’s Bloomington, Indiana plant has received an FDA Form 483 after an agency inspection of the site in late June and July. The Form 483 has flagged serious quality lapses at the Indiana plant — a site it acquired through its US$ 16.5 billion acquisition of Catalent last year. Inspectors cited contamination risks ranging from cat hair to bacterial incursions, along with pest issues and repeated equipment failures.

Regeneron’s experimental drug for myasthenia gravis scores phase 3 win

Regeneron’s new drug cemdisiran has shown promising results in patients with generalized myasthenia gravis (gMG), a rare disease that weakens muscles. In a 24-week study, patients who received cemdisiran, a small RNA therapy injected under the skin once every three months, reported clear improvements in daily function. Regeneron plans to seek FDA approval in 2026.

Lilly’s oral GLP-1 pill reduces 10.5% weight in phase 3 trial: Eli Lilly’s oral pill orforglipron helped people with type 2 diabetes and excess weight lose an average of 10.5 percent of body weight in a 72-week phase 3 trial. The pill also improved blood sugar levels. Analysts said the results matched expectations and should support an FDA submission this year.

Akeso, Summit’s ivonescimab extends life in phase 3 lung cancer trial

Akeso and Summit Therapeutics said their experimental cancer drug ivonescimab helped patients suffering from EGFR-mutated non-small cell lung cancer (NSCLC) live longer in a late-stage study. The phase 3 trial was held in China. This is the first time the medicine has shown that it can extend life, and not just slow disease progression.

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