Welcome to the Weekly Rundown where the DDN  editors cover this week’s top biotech and pharma news.

FDA announces new voucher program to speed up drug review

On Tuesday, the FDA announced a new priority review system called the Commissioner’s National Priority Voucher (CNPV) program that aims to shorten the drug review process from 10–12 months down to just one to two months. Their announcement detailed plans to provide vouchers to companies that are “aligned with U.S. national priorities,” including those that address a health crisis, provide more innovative cures, address unmet needs in public health, and increase drug manufacturing in the US. To describe how the new process works, FDA Commissioner Martin Makary said in the press release, “As a surgical oncologist, we often made multidisciplinary decisions with a team of doctors on major life-and-death questions for patients, incorporating the latest medical studies in a 1-day tumor board-style discussion. This voucher harnesses that model to deliver timely decisions for drug developers.” – Allison Whitten

Late-stage setback for AbbVie’s blood cancer drug

AbbVie has announced that its blood cancer drug Venclexta did not meet the primary endpoint in a Phase 3 trial for higher-risk myelodysplastic syndromes (MDS) — a group of cancers where the bone marrow fails to produce enough healthy blood cells. The VERONA study, which tested Venclexta in combination with azacitidine in newly diagnosed MDS patients, showed no significant improvement in overall survival compared to standard treatment. While this outcome is a setback for expanding the drug’s use, AbbVie confirmed it does not affect Venclexta’s existing approvals, including its use in certain leukemias. Already generating $2.6 billion in global sales in 2024, Venclexta remains a key asset in AbbVie and Roche’s oncology pipeline, with ongoing trials in other blood cancers like non-Hodgkin lymphoma and multiple myeloma. Full data from the VERONA trial will be shared at a future medical meeting. – Bree Foster

Duchenne muscular dystrophy gene therapy leads to second death

Sarepta Therapeutics’ gene therapy to treat Duchenne muscular dystrophy, Elevidys, led to the second death of a patient due to liver failure. The company announced that they have temporarily stopped shipments of Elevidys for non-ambulatory patients, and paused the ENVISION study while they work on adding a protocol amendment that will enhance the immunosuppression regimen to moderate liver enzyme elevations. Over 900 individuals have already been treated with Elevidys. – Allison Whitten

FDA approves first-of-its-kind treatment for hereditary angioedema

CSL has received FDA approval for Andembry, a new preventive treatment for hereditary angioedema (HAE) — a rare and potentially life-threatening genetic disorder that causes unpredictable and painful swelling attacks. Andembry is the first and only treatment that blocks factor XIIa, a key protein that triggers these attacks. Delivered as a once-monthly at-home subcutaneous injection, Andembry was shown in the Phase 3 VANGUARD trial to dramatically reduce HAE attacks by over 99 percent in the median case, with 61.5 percent of patients remaining attack-free for the entire six-month trial. Andembry is the first approved drug to inhibit the HAE pathway at its source rather than targeting downstream effects, providing a simpler and more effective option for people living with HAE. – Bree Foster

New blood test could prevent organ rejection of liver transplants

After receiving a life-saving liver transplant, many patients’ immune systems unfortunately reject the new organ and attack it as foreign. Researchers at Georgetown University and MedStar Health developed a new blood test that could alert doctors to early signs of organ rejection, which could allow for personalized treatments to prevent organ failure (1). By analyzing cell-free DNA fragments released from dying liver cells into the blood, the team found signatures that suggest early-onset injury of the transplant. In a press release, study author Anton Wellstein said, “It was amazing how well it worked.” – Allison Whitten

References

1. McNamara, M.E. et al. Circulating cell-free DNA methylation patterns indicate cellular sources of allograft injury after liver transplant. Nat Commun 16, 5310 (2025).