December 17, 2025

The need-to-know this morning

Cytokinetics said Chinese regulators approved Myqorzo (formerly aficamten) for the treatment of a serious heart condition known as obstructive hypertrophic cardiomyopathy. An approval decision from the FDA is expected before the end of the year. Sanofi has rights to sell Myqorzo in China.
DBV Technologies said its Viaskin peanut patch reduced allergic reactions in children, achieving the primary goal of a Phase 3 clinical trial. The company plans to submit for FDA approval next year.

science

Some scientists are leaving the U.S. as Trump policies destabilize research prospects

The volatility of federal research funding under the Trump administration has stoked fears of a U.S. brain drain. While it's too early to know how many scientist have left or will leave the country this year, there are some signs that more researchers are looking abroad.

The European Research Council, a funding body, has seen the number of applicants for early-career grants from the U.S. nearly triple in recent years, rising from 60 for the 2024 call, to 116 for 2025, to 169 for 2026. For grants for more senior researchers, the number of applicants from the U.S. rose from 23 for the 2024 call to 114 this year.

STAT's European correspondent Andrew Joseph spoke to a half-dozen scientists who have left or in the process of leaving. In some cases, they said they no longer wanted to be subjected to the uncertainty under Trump's policies; in others they could no longer find work in the U.S. as universities and labs grapple with funding cuts and grant freezes.

biotech

Who was the worst biotech CEO of 2025?

Yesterday, we gave you my colleague Adam Feuerstein's list of the best CEOs of the year. Today, we have his pick for worst CEO: Sarepta Therapeutics' Doug Ingram.

His decision centers around Ingram's move to aggressively push for the broadest possible approval for Sarepta's gene therapy for Duchenne muscular dystrophy, including in older boys who lost the ability to walk, despite scant clinical data.

This year, two teenage boys died from acute liver failure after taking the therapy, Elevidys. An older man also died from liver failure taking an experimental Sarepta gene therapy that uses the same engineered virus as Elevidys to transport the gene.

Read more on the reasoning behind Adam's pick — and on his runners-up.

pharma

A much-needed win for Pfizer

From STAT's Matthew Herper: Pfizer said this morning that giving its antibody-drug conjugate, Padcev, in combination with Keytruda before and after surgery extended survival in a clinical trial compared to giving two chemotherapies, gemcitabine and cisplatin. The results add to earlier positive data that will be reported in August for the drug, which came from Pfizer’s $43 billion purchase of Seagen.

The results come a day after Pfizer trimmed its sales and earnings guidance for 2025 and 2026, leading shares to drop 3.5%. The sources of the pressure: lower Medicaid prices as a result of the deal the drug giant negotiated with the Trump administration and lower sales of Covid vaccines.

As far as the anti-vaccine backlash in the U.S., Pfizer CEO Albert Bourla told investors on a conference call that doubts about vaccines go against science that dates back to the time of Louis Pasteur. “We will continue investing in vaccines because, as I said, this is an anomaly that will correct itself,” Bourla said.

policy

HHS recommends screening newborns for two forms of dystrophy

Health secretary Robert F. Kennedy Jr. yesterday added two rare diseases, Duchenne muscular dystrophy and metachromatic leukodystrophy (MLD), to the list of conditions the government recommends states screen newborns for.

The move came eight months after the Department of Health and Human Services disbanded the expert committee that was set to vote on adding these diseases to the screening recommendations.

Advocates greeted the move as a critical step toward enabling earlier diagnosis, which is even more important now as companies develop more therapies for the diseases.

politics

RFK Jr. is consolidating power while maintaining Trump's support

In the months since RFK Jr. took the helm of HHS, he's carried out major reorganizations of the agencies under him, effectively cementing his power.

He reconstituted a key panel of federal vaccine advisers with allies and vaccine skeptics, and he's overseen drastic cuts to funding for health research.

At the same time, Kennedy has continued to keep a warm relationship with President Trump, while Congress has faded into the background. Congress is supposed to approve most major departmental changes, but staffers say that get little information from HHS about its plans.