Experts review muscular dystrophy treatment

May 12, 2023

Presented by The Campaign for Sustainable Rx Pricing

With help from Alice Miranda Ollstein and Robert King

Driving the day

A sign for the Food And Drug Administration is seen outside of the headquarters.

Drugmaker Sarepta seeks accelerated approval from the FDA for its gene therapy for Duchenne muscular dystrophy. | Sarah Silbiger/Getty Images

ADVISERS WEIGH CONTENTIOUS GENE THERAPY — The FDA’s independent experts meet today to consider the first gene therapy for a fatal neuromuscular condition.

Duchenne muscular dystrophy is a rare genetic condition that prevents patients from producing a usable form of a protein called dystrophin, which is vital to muscle function. Sarepta Therapeutics, the treatment’s developer, says its therapy could help make the condition milder. But FDA scientists who reviewed the company’s data appear not convinced.

The disease primarily affects boys and causes progressive weakness and heart complications that are ultimately fatal. About 1 in 3,600 boys are born with the condition each year, and average life expectancy is between 20 and 30 years old.

FDA concerns: Sarepta seeks accelerated approval for the drug candidate, but in published briefing documents, agency scientists concluded the data doesn’t seem to support the application: Roughly a year after treatment, patients in a randomized trial didn’t have functional improvements compared with those who received a placebo.

The agency also raised concerns about the drug’s safety, especially the risk of patients developing cancer. And Sarepta had to temporarily pause its trial in August 2021 when a patient who received treatment needed to be hospitalized after developing severe asthma.

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TODAY ON OUR PULSE CHECK PODCAST , host Ruth Reader talks with Megan Messerly about the Democratic lawmakers in some states who are hesitant to expand health coverage to low-income undocumented immigrants because of budgetary concerns, leading some immigration advocates wondering how committed to the issue those Democrats really are.

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Big Pharma’s price gouging is the root cause of out-of-control drug prices. Nearly half of all brand name Rx drugs now cost more than $150,000 per year when they are brought to market. It’s time to hold Big Pharma accountable. Learn more.

In Congress

Senate Majority Leader Chuck Schumer standing during a press conference at the U.S. Capitol.

Sen. Chuck Schumer wants the Senate to pass a health package this month. | Francis Chung/POLITICO

HELP ADVANCES PBM, GENERICS PACKAGE — In a broad bipartisan vote, the Senate HELP Committee passed a legislative package Thursday intended to rein in pharmacy benefit managers — the pharmaceutical middlemen that manage prescription drugs for health insurers — and boost generic drug competition.

The second successful attempt to move the bills to the Senate floor comes after a previous attempt was postponed when lawmakers alleged committee Chair Bernie Sanders (I-Vt.) did not honor amendment agreements made during negotiations with ranking member Bill Cassidy (R-La.). The successful markup comes after more than a week of negotiations aimed at advancing a package the two say can pass the upper chamber.

It is unclear when a floor vote will be scheduled, said Sanders and Cassidy. But Sen. John Hickenlooper (D-Colo.) told POLITICO he expects a vote to be held in “weeks rather than months,” and Senate Majority Leader Chuck Schumer (D-N.Y.) indicated he wants the Senate to pass a health package in May.

INSULIN IN THE HOT SEAT — Insulin maker and pharmacy benefit manager executives stuck to their scripts at a separate Senate HELP Committee hearing this week, blaming each other for a complicated pricing system that lawmakers argue hurts diabetes patients.

Despite some praise from senators for recent commitments by drugmakers to lower certain insulin list prices, the upper chamber is poised to move forward legislation to limit out-of-pocket costs for those with private insurance.

Sen. Susan Collins (R-Maine) told POLITICO on Thursday she expects insulin legislation to be added to the drug pricing package passed out of the HELP Committee on the Senate floor.

WYDEN SLAMS PHARMA TAX STRATEGIES — Billions of dollars in taxable drug sale revenue is still being taxed as offshore income by pharmaceutical manufacturers, according to a Senate staff report that Democrats say demonstrates the need to do more to make drugmakers pay their fair share of taxes.

Senate Finance Committee Chair Ron Wyden (D-Ore.) highlighted Merck’s taxation strategy for its blockbuster cancer drug Keytruda, which generated $37 billion in sales from 2019 through 2022. The report found that nearly all the income from such sales was taxed as offshore income, Robert reports.

Merck said in a statement to POLITICO that the company complies with the laws of each jurisdiction that it operates in and its global income is subject to the U.S. global minimum tax rate. The company also credited the 2017 tax cuts with allowing U.S. companies to be more competitive, said spokesperson Robert Josephson.

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Eye on the FDA

FDA EASES BLOOD DONATION RESTRICTIONS — The FDA finalized plans on Thursday to loosen blood donation restrictions for men who have sex with men. Some men in monogamous relationships with another man can forgo the three-month abstinence period previously required and take a questionnaire instead. The FDA said it “strongly believes” the updates won’t harm the nation’s blood supply.

Some restrictions still apply: Screening questions asked of prospective blood donors will still recommend the deferral of individuals who report having a new sexual partner and have engaged in anal sex in the past three months, as well as of individuals who report having more than one sexual partner in the last three months and have also had anal sex.

The policy also limits blood donation for people who have used intravenous nonprescription drugs, have exchanged sex for money, have been diagnosed with HIV or are taking pre-exposure prophylaxis or post-exposure prophylaxis against HIV.

FDA APPROVES NEW REXULTI INDICATION — Rexulti, a drug used to treat schizophrenia, major depressive disorder and psychosis, received FDA approval to treat agitation related to dementia on Thursday.

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Industry Intel

CVS PLEDGES TO STOCK OTC BIRTH CONTROL — The country’s biggest retail pharmacy chain will stock the over-the-counter birth control drug Opill in its more than 10,000 U.S. stores if the FDA approves it for sale sometime this summer.

“We want to be a premier health and wellness destination,” Prem Shah, CVS’ chief pharmacy officer, told Alice on Thursday. “So if it’s available over the counter as something that could be utilized, we would absolutely provide it as long as legally enforceable.”

Shah said the company has no plans for individual locations to opt out of selling the drug.

Sen. Patty Murray (D-Wash.) applauded the adviser vote and encouraged the FDA to follow their recommendation. “The fight to expand access to contraception is even more important today in a world without Roe,” she said Thursday.

This week, two independent agency advisory panels unanimously endorsed the FDA’s approval of Opill.

WHITE HOUSE LOOKING INTO DRUG SHORTAGES — The White House is working to identify policies that could help improve persistent supply-chain issues and drug shortages, a former FDA official and a senior administration official granted anonymity to discuss the effort, confirmed.

The effort, first reported by Bloomberg, aims to strengthen pharmaceutical supply chains so Americans can obtain drugs when they need them, according to the senior administration official. The former FDA official said White House staff are frustrated with the FDA partly because the agency does “not appear to be in front of this issue.”

“The agency is fully engaged in cross-government discussions to identify potential policies and provide input on proposals informed by our current authorities, resources and industry knowledge to ensure meaningful solutions,” FDA spokesperson Michael Felberbaum said in an email.

At a House Energy and Commerce Health Subcommittee hearing Thursday on public health threats, FDA Commissioner Robert Califf said low margins for generic manufacturers are contributing to drug shortages.

“We’ve got to deal with these adverse economics that are occurring, those are well beyond the FDA’s remit, but it’s an important part” of the problem, Califf said.

Anthony Sardella, chair of the API Innovation Center, a nonprofit focused on drug supply-chain issues, told the Oversight and Investigations Subcommittee on Thursday that incentivizing domestic drug manufacturing needs to be part of the solution.

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QUICK HIT

In JAMA Internal Medicine this week, researchers estimate that Medicare will spend between $2 billion and $5 billion annually if it fully covers Leqembi, a novel Alzheimer’s treatment.

A message from The Campaign for Sustainable Rx Pricing:

In Big Pharma’s game, out-of-control drug prices win and patients lose. Big Pharma’s anti-competitive practices will cause patients to spend $167 BILLION over seven years on just three branded drugs. It’s time to hold Big Pharma accountable. Learn more.