Who gets to benefit from the first CRISPR cures?

In clinical trials, a CRISPR-based treatment for sickle cell disease has liberated patients from the agonizing pain crises that once constrained their lives. The first such treatment, from Vertex Pharmaceuticals and CRISPR Therapeutics, could win FDA approval later this year. But getting the therapy to sub-Saharan Africa, where the majority of the world's sickle cell patients live, poses massive challenges.

aaAs STAT's Megan Molteni reports, the price of CRISPR medicines could mirror the $4 million to $6 million a single sickle cell patient in the U.S. accrues in costs over a lifetime of drugs, infusions, and hospital visits. Beyond the cost, low-income countries often lack the clinical infrastructure necessary for such cutting-edge treatments, which require bone marrow to be collected, edited, and then infused back into patients.

"The road from now, having gene editing that is possible and implemented where most people live, it's not that close," said Ambroise Wonkam, a geneticist at Johns Hopkins School of Medicine and the University of Cape Town who also serves as president of the African Society of Human Genetics. "We have to put that into perspective."

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Financials

BioMarin's push for profitability hits a snag

The FDA again delayed a long-awaited approval for BioMarin's gene therapy for hemophilia, further prolonging the medicine's U.S. launch and potentially affecting the company's goal of finally becoming a consistently profitable enterprise.

The news, disclosed yesterday, is that the FDA is taking another three months to review Roctavian, a one-time treatment for hemophilia A. The new decision date is June 30. The delay was always a possibility, after the company submitted another tranche of safety and efficacy data last year to support Roctavian's approval.

Just last week, BioMarin forecast 2023 sales of its gene therapy of between $100 million and $200 million. The FDA's decision could cut into those projections and make BioMarin's goal of turning a roughly $200 million profit this year difficult to reach.

China

The Sino-American biotech era isn't quite over

After months of deliberation, U.S. regulators finally approved the merger of a domestic biotech company with a Chinese pharma firm, a ruling that could have broader implications for the industry.

The Committee on Foreign Investment in the United States, or CFIUS, signed off on Sino Biopharmaceutical's $161 million buyout of F-Star Therapeutics, the companies said yesterday, clearing a deal that has gone through more than a dozen amendments since its announcement in June 2022. Over the past five years, the U.S. government has blocked a handful of biotech transactions in the name of national security, leading to a chill in cross-border deals with Chinese investors and drug developers.

Biotech companies with operations in the U.S. and China have seen their valuations dip since President Xi Jinping tightened his control of the country in October. The underlying fear is that Xi's third term in power will escalate state control at the expense of the private sector, which, combined with the White House's efforts to reduce the U.S.'s dependence on China, will leave Sino-American biotech companies caught in the middle.

Regulatory

Covis blinked in its FDA standoff

Covis Pharma, maker of a treatment meant to prevent preterm births, has agreed to follow the FDA's directive and pull its drug from the market.

As STAT's Ed Silverman reports, Covis said in a letter to the agency that while it stands by its drug's benefits, the company "has made the very difficult decision to voluntarily withdraw" it. The decision comes after a panel of FDA advisers voted 14-1 in favor of revoking the treatment's approval in October.

The drug, called Makena, won accelerated approval in 2011 based on preliminary evidence but failed to show any benefit in a confirmatory study disclosed in 2019, leading the FDA to call for its removal from the market. Covis contested that decision, mounting a challenge to the FDA's authority that has now come to an end.

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