What the withdrawal of Pfizer's sickle cell drug means

A day after Pfizer pulled its sickle cell drug Oxbryta from the market due to safety concerns, patients and advocates are expressing shock.

The news is an embarrassment for the drugmaker, which spent $5.4 billion to purchase Global Blood Therapeutics, which developed the therapy, in 2022. But there are also questions about whether pulling the drug is the right choice.

"We have so few treatment options," one pediatric hematologist told STAT. "Could there not be a published warning, even a 'black box warning,' instead of the unusual step of taking away the medicine from everybody with sickle cell disease?"

The drug was approved in 2019, but it was a controversial one — with questions swirling even then about its safety and efficacy.

pricing

CMS drops Medicaid drug price transparency plan

The Biden administration just scrapped a proposed policy that would have mandated drugmakers disclose the prices and research costs for high-cost medicines in state Medicaid programs. The decision comes after intense lobbying and legal threats from pharma companies, which saw the proposal as an overreach of government authority.

Ultimately, STAT's Bob Herman writes, the reversal highlights how the Biden administration was not willing to take on a unified drug industry that is still separately attacking the government's Medicare drug negotiation program.

BIOTECH

Cassava Sciences and former execs, facing SEC charges, agree to fines

Facing charges from the Securities and Exchange Commission for misleading claims related to a controversial Alzheimer's drug candidate, Cassava Sciences and two of the company's former executives have agreed to pay fines, the company announced yesterday.

The news came on the same day that the SEC filed charges against Cassava, former CEO Remi Barbier, and former senior vice president of neuroscience Lindsay Burns.

mental health

FDA approves Cobenfy as new schizophrenia treatment

U.S. regulators yesterday approved Cobenfy — a new type of schizophrenia treatment developed by Karuna Therapeutics. It'll be sold by Bristol Myers Squibb.

The drug has a mechanism of action that's unique to other psychiatric medicines used to treat schizophrenia: It stimulates the muscarinic receptors in the brain to help reduce psychotic symptoms, STAT's Adam Feuerstein writes.

Trials show that Cobenfy may improve cognition and treat negative symptoms like social withdrawal. This novel approach has fewer side effects, such as weight gain, which is a major issue with other medications. It'll cost $22,500 each year, and some analysts project that sales could grow to $3.4 billion annually.

podcast

Why are Genentech cancer researchers worried?

What led Pfizer to pull its sickle cell drug off global markets? And what cake did Adam make for all his podcast colleagues except Elaine? We talk about all that and more on this week's episode of "The Readout LOUD," STAT's biotech podcast.

Our colleague Jonathan Wosen joins us to discuss the recent setbacks and wins in oncology R&D, including Genentech's decision to shut down its cancer immunology group, and the global ambitions of Akeso, the Chinese biotech behind Summit Therapeutics' recent positive results.

Listen here.

gene therapy

Dispute over Duchenne therapy highlights thorny access issues

Since the FDA granted wide approval to Elevidys, a gene therapy for Duchenne muscular dystrophy developed by Sarepta Therapeutics, families seeking access for their children have reported challenges.

The latest case in point: 15-year-old Sarah Jenssen, who lives in Alabama.

Though the FDA authorized the treatment for patients who rely on wheelchairs, as Sarah does, her insurer denied her access. Only last week, following appeals by her medical team, did it reverse course.

"You spend all this time to get the FDA to approve it, and then you have to spend even more time with insurance companies to get them to approve it," one of her doctors told STAT's Andrew Joseph.

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