For rare disease families, gene therapy comes with a deadline

The long-awaited approval of the first gene therapy for Duchenne muscular dystrophy, made by Sarepta Therapeutics, came with a catch: The one-time treatment is approved only for kids aged 4 or 5. That has left physicians and families scrambling to get access to the costly medicine before their eligible patients turn 6.

As STAT's Jason Mast reports, receiving Sarepta's gene therapy, Elevidys, is not as simple as taking a prescription to a pharmacy. Only a handful of neuromuscular centers around the country can administer the treatment, and that's only after they've properly screened patients, secured insurance for the $3.2 million therapy, and obtained the actual drug product. For the eldest among eligible 5-year-olds, it's a tight window.

The restrictive label reflects Sarepta's supporting data. A clinical trial showed dramatic benefits for 4- and 5-year-olds, but not for 6- and 7-year-olds. But what vexes physicians are the marginal cases. Is a patient who is 5 years and 11 months really all that different from one who is 6 years and a day? "It's arbitrary," said Emma Ciafaloni, a neuromuscular specialist at Rochester University who treats Duchenne. "Very, very arbitrary."

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Moderna is trading like its Covid vaccine never happened

D3 vis exported to PNG (66)

Shares of Moderna dipped below $100 yesterday, hitting their lowest value since November 2020, back when no one knew whether the company's in-development vaccine for Covid-19 would succeed in pivotal trials.

Nearly three years and $40 billion in revenue later, Wall Street seems to think the company's best days have passed. Only 10 of 22 analysts maintain a buy rating on Moderna's stock, according to StreetAccount. That's despite the company losing nearly 50% of its value in 2023 alone.

Moderna's near-term fortunes could still change if there's a spike in demand for Covid booster shots in the fall, which would ease the pressure on the company's balance sheet as it looks forward to potential product launches in 2024 and beyond. But if sales continue their decline, Moderna "is a likely candidate for restructuring and headcount reduction," Leerink analyst Mani Foroohar wrote in a recent note downgrading the company's shares.

Marketing

The FDA is close-reading pharma's collateral

The FDA admonished AstraZeneca for making a misleading claim about a medicine for COPD, issuing a rare warning letter after years of declining enforcement.

As STAT's Ed Silverman reports, at issue is a promotional sales aid suggesting AstraZeneca's treatment, called Breztri, reduced the risk of death in COPD patients. That claim isn't supported by the study cited in the brochure, according to the FDA, which called AstraZeneca's marketing materials "concerning from a public health perspective."

The letter is part of what appears to be a trend at the FDA's Office of Prescription Drug Promotion. While the department has issued few warnings in recent years, the last four have related to efficacy claims that weren't backed up by cited data, suggesting the agency is taking a harder look at pharma's marketing to physicians.

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