Experts Consider Treatment for Muscular Dystrophy


With the help of Alice Miranda Allstein and Robert King

Advisors Consider Controversial Gene Therapy — Independent FDA experts meet today to explore the first gene therapy for a deadly neuromuscular disease.

Duchenne muscular dystrophy is a rare genetic disorder that prevents patients from producing a usable form of a protein called dystrophin that is essential for muscle function. Sarepta Therapeutics, which developed the treatment, says it may help reduce symptoms. But FDA scientists who have looked at the company’s data don’t seem convinced.

The disease primarily affects boys and can lead to progressive weakness, heart complications, and ultimately death. About 1 in 3,600 boys are born with this condition each year, and the life expectancy is 20-30 years for him.

FDA concerns: Sarepta is seeking early approval for the drug candidate, but in a public explanatory document, agency scientists concluded that the data did not appear to support the application: about a year after treatment, Patients in randomized trials received placebo, which showed no improvement in function compared to treated patients.

The agency also expressed concerns about the drug’s safety, particularly about the risk of patients developing cancer. Sarepta was forced to suspend the trial in August 2021 after a patient undergoing treatment developed severe asthma and required hospitalization.

thatFriday. Welcome to Prescription Pulse. Happy Zelda Day to those who celebrate.

Send news, tips, and Tears of the Kingdom thoughts to David Lim ([email protected] again @Devidalim) or Catherine Ellen Foley ([email protected] again @katherineefoley).

today our pulse check podcast , Host Ruth Reeder on Megan Messary and Democrats in some states hesitant to expand medical coverage for low-income illegal immigrants due to budget concerns, some pro-immigrant Democrats I wonder how serious lawmakers are on this issue.

Help us advance PBM, generic packaging — The Senate Support Committee on Thursday voted in a broad bipartisan vote aimed at curbing pharmacy benefits administrators (pharmaceutical brokers who control prescriptions for health insurance companies) and promoting generic drug competition. passed the bill.

The second attempt to move the bill to the floor of the Senate saw Chairman Bernie Sanders (Ime, Vermont) amend the agreement reached during negotiations with Rep. Bill Cassidy (Republican, Laminate). This was after a previous attempt was postponed, alleging non-compliance. ). The successful price hike comes after more than a week of negotiations aimed at advancing a package that the two men claim can pass the Senate.

Sanders and Cassidy said it was unclear when a congressional vote would be scheduled. But Sen. John Hickenlooper The (Democratic Party of Colorado) told POLITICO it expected the vote to take place in “weeks, not months,” and the Senate Majority Leader said: Chuck Schumer (DN.Y.) said he hopes the Senate will pass a health policy in May.

hot seat insulin —Insulin makers and pharmacy benefits administrator executives stuck to their script at another Senate Support Committee hearing this week, arguing lawmakers are hurting diabetics blamed each other for their complicated pricing system.

Recent efforts by pharmaceutical companies to lower the price of certain insulins have drawn some praise from senators, but the Senate is poised to push forward with legislation to limit out-of-pocket costs for private insurers.

Sen. Susan Collins (R. Maine) told POLITICO on Thursday that she expects the insulin bill to be added to the drug pricing package passed by the HELP committee on the Senate floor.

Wieden slams drug tax strategy — Billions of taxable drug sales revenue is still taxed as offshore income by drug makers, says Senate staff report, and Democrats need to do more to get drug makers to pay their fair share of taxes It states that it is necessary.

Chairman of the Senate Finance Committee Ron Wyden (Democrat, Oregon) highlighted Merck’s tax strategy for blockbuster cancer drug Keytruda, which generated $37 billion in sales from 2019 to 2022. The report found that nearly all of the income from such sales was taxed as offshore income, Robert reports.

In a statement to POLITICO, Merck said it complies with the laws of each jurisdiction in which it operates and that its global profits are subject to the US global minimum tax rate. Company spokesman Robert Josephson also said the company believes the 2017 tax cuts have made U.S. companies more competitive.

FDA relaxes restrictions on blood donation — The FDA on Thursday finalized a plan to ease blood donation restrictions for men who have sex with men. Some men in monogamous relationships with other men forgo the previously required three-month period of abstinence and take surveys instead. The FDA said it “strongly believes” that the update will not adversely affect the public’s blood supply.

Some restrictions still apply. Screening questions asked of prospective blood donors included those who reported having a new sexual partner and reported participating in anal sex within the past 3 months, and those who reported having had multiple sexual partners in the past 3 months. We continue to recommend deferrals to those who reported them. We had anal sex for several months.

The policy also includes those who have used intravenous non-prescription drugs, exchanged money for sex, been diagnosed with HIV, or received pre-exposure or post-exposure prophylaxis for HIV. restrict blood donation to those who

FDA Approves New Indication for Rexulti — Rexulti, a drug for treating schizophrenia, major depressive disorder and psychosis, received FDA approval on Thursday for the treatment of dementia-related agitation.

CVS Commits to Stock Over-the-Counter Birth Control — The country’s largest retail pharmacy chain plans to stock over-the-counter contraceptive Opil in more than 10,000 U.S. stores if the FDA approves it for sale later this summer.

“We want to be the premier destination for health and wellness,” CVS Chief Pharmacy Officer Prem Shah told Alice on Thursday. “So if it’s available over the counter as available, we’re absolutely going to offer it as long as it’s legally enforceable.”

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

Sen. Pattie Murray (Washington Democrat) applauded advisory vote and encouraged the FDA to follow its recommendations. “Today, in a world without contraceptives, the fight to expand access to contraception is even more important. egg‘ she said on Thursday.

This week, two independent advisory committees voted unanimously to support the FDA’s approval of Opill.

White House investigating drug shortage — The White House acknowledges it is working to identify policies that can help ameliorate persistent supply chain problems and drug shortages, and has allowed a former FDA employee and senior government official to discuss the effort on condition of anonymity. rice field.

The effort, first reported by Bloomberg, aims to strengthen the drug supply chain so that Americans can get drugs when they need them, senior government officials said. A former FDA official said White House officials were unhappy with the FDA in part because the agency “seems to be failing to address the issue head-on.”

“To ensure meaningful solutions, FDA is committed to intergovernmental discussions to identify potential policies and provide input on proposals drawn from current agency, resources, and industry knowledge. FDA spokesman Michael Felberbaum said in an email.

At a hearing of the House Energy, Commerce and Health Subcommittee on Public Health Threats on Thursday, FDA Commissioner Robert Calif said low profit margins at generic manufacturers are contributing to drug shortages. .

“We have to deal with the economic ramifications that are happening now.

Anthony Sardera, president of the API Innovation Center, a nonprofit focused on pharmaceutical supply chain issues, told a monitoring and investigation subcommittee on Thursday that encouraging domestic drug manufacturing must be part of the solution. rice field.

In this week’s issue of JAMA Internal Medicine, researchers estimate that if Medicare had full coverage for the new Alzheimer’s treatment, Rekumbi, it would cost $2 billion to $5 billion annually.





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