First CRISPR therapy one step closer to approval

Oct 31, 2023

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By Lauren Gardner, David Lim and Katherine Ellen Foley

Presented by

Coalition for Affordable Prescription Drugs

Driving the Day

A laboratory staffer works on gene therapy

FDA advisers will examine evidence regarding the risk potential of a first-of-its-kind therapy to treat sickle cell disease. | Eric Piermont/AFP via Getty Images

A GENE-EDITING BREAKTHROUGH — Expert advisers will consider today how two companies assessed potential risks with their proposed first-ever gene-editing treatment for sickle cell disease, a rare condition that disproportionately affects people of color.

The therapy uses a groundbreaking gene-editing technique known as CRISPR to modify the stem cells of patients with the disease and is in line to be the first such treatment approved for sickle cell disease. An FDA decision is expected by Dec. 8.

The companies behind the candidate drug, Vertex Pharmaceuticals and CRISPR Therapeutics, have also applied for the therapy to be indicated for another rare blood condition, transfusion-dependent beta thalassemia.

Up for discussion: “It is unclear whether the analysis using this limited sample size will provide for an adequate understanding of the potential risk of off-target editing,” a phenomenon in which the therapy edits a gene that isn’t the focus of the treatment, FDA reviewers said in a briefing document released ahead of the meeting.

The agency is asking members of its independent advisory committee on cell, tissue and gene therapies to discuss whether the companies’ off-target analysis was adequate to evaluate risk and whether more studies are warranted.

The big question is whether FDA advisers will favor additional studies, and if so, whether they should be conducted before or after approval. The discussion topic isn’t subject to a vote, so the agency will have no formal recommendation to consider.

Still, the debate will inform the FDA’s decision on whether the treatment, dubbed exa-cel, should be approved this year.

Investment banking firm William Blair called the briefing document “a near-best-case scenario” for the companies in a Friday research note, nodding toward the treatment’s status as the first CRISPR-based therapy to be considered for FDA approval.

The other looming issue is how or whether insurers would cover the treatment if approved. It likely would be available to anywhere from 10,000 to 20,000 sickle cell patients with severe disease — a much larger patient pool than for other rare diseases with FDA-approved gene therapies, according to Stephen Majors of the advocacy group Alliance for Regenerative Medicine.

Gene therapies are typically intended to be curative, meaning their one-time or limited use can command million-dollar price tags. The nonprofit Institute for Clinical and Economic Review found that exca-cel and bluebird bio’s genetic treatments for sickle cell disease would be cost-effective between $1.35 million and $2.05 million.

IT’S TUESDAY. WELCOME BACK TO PRESCRIPTION PULSE. Congratulations to our Katherine Ellen Foley for finishing the Marine Corps Marathon! It was a brutal weather day for a 26.2 mile race; we’re so proud of her!

Send news, tips and cool weather vibes to Lauren Gardner (lgardner@politico.com or @Gardner_LM), David Lim (dlim@politico.com or @davidalim) and Katherine Ellen Foley (kfoley@politico.com or @katherineefoley).

TODAY ON OUR PULSE CHECK PODCAST, host Chelsea Cirruzzo talks with POLITICO health care reporter Ben Leonard, who breaks down President Joe Biden's executive order directing HHS to develop guidelines and oversight for the use of AI in health care and explains the order's potential impact.

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A message from Coalition for Affordable Prescription Drugs:

A new survey shows 97% of employers who contract directly for pharmacy benefit services are satisfied with their PBM. And 86% of employers say it is important to have a range of options in how they pay PBMs for their services and expertise. Read the survey results.

A bottle of Eliquis is shown.

Eliquis, used to treat and prevent blood clots, was the first drug among those involved in Medicare drug price negotiations to undergo a public listening session. | Scott Olson/Getty Images

CMS KICKS OFF PATIENT-LISTENING SESSIONS — Bristol Myers Squibb’s blood-clotting drug Eliquis was the first of the 10 medications selected for Medicare drug price talks to be the focus of a public listening session held by CMS on Monday — a multiweek process that continues today with Amgen’s rheumatoid arthritis drug Enbrel.

White House press secretary Karine Jean-Pierre told reporters she will highlight each drug selected for Medicare negotiations at briefings over the coming days, a sign of the administration’s belief that the landmark pricing negotiations represent a keen political messaging opportunity ahead of the 2024 presidential election.

On Monday, she highlighted what patient and consumer advocates said at the first CMS listening session. “Lowering costs for each of these drugs will … save lives,” Jean-Pierre said.

Sarah Ryan, spokesperson for the pharmaceutical industry trade group PhRMA, slammed the listening sessions as simply a “box-checking exercise” with limited utility because CMS will not respond to feedback in real time.

But Dr. Meena Seshamani, director of CMS’ Center for Medicare, told participants at the listening session that they are meant to be a transparent public engagement process that will provide CMS with input on the 10 drugs selected for the first negotiations.

“Our priority in negotiating with participating drug companies is to come to an agreement on a fair price for Medicare,” Seshamani said.

Tobacco

A CTP UPDATE — The nation’s top tobacco regulator has made progress on shoring up a policy office within its Office of the Center Director, according to its director, Brian King, in a centerwide update Friday.

The update, published in response to last year’s Reagan-Udall Foundation evaluation of tobacco regulation, noted that the Center for Tobacco Products had published a one-page printout of all the e-cigarette products legally allowed on the market for retailers and is working on a centralized database to search for authorized products.

King alluded to additional details to be shared in a strategic plan for the center later this year. “Some of the planned actions require more time, and contributions outside of the center, than others, but we continue to make strides in all areas to put our blueprint into action,” King said.

He reiterated that the CTP seeks $100 million in additional user fees from cigarette makers and the ability to collect fees from e-cigarette manufacturers, a goal it has pursued since 2020. Despite taking up most of the CTP’s time and resources, e-cigarette manufacturers don’t pay user fees — which some policy experts say contribute to delays in new product authorization reviews.

“I really do question where this money is going … essentially, it’s a blank check,” said John O’Brien, vice president and assistant general counsel for Reynolds American, a subsidiary of British American Tobacco and parent company of R.J. Reynolds.

FTC: TOBACCO SALES DROP SLIGHTLY — Large cigarette companies sold 16.7 billion fewer cigarettes to wholesalers and retailers in 2022 than in 2021, according to the Federal Trade Commission’s annual cigarette report. Menthol-flavored cigarettes, which the FDA plans to ban later this year, represented 36 percent of the 173.5 billion cigarette market in 2022.

A message from Coalition for Affordable Prescription Drugs:

WHAT WE'RE READING

The Biden administration is calling on schools to stock naloxone to combat opioid overdoses as teen deaths rise, POLITICO’s Mackenzie Wilkes reports.

Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, Elevidys, did not improve muscle function against a placebo in a large trial, STAT’s Jason Mast and Adam Feuerstein report.

Pharma Moves

The Hearing Industries Association announced Bridget Dobyan will take over as its executive director at the start of 2024.

Families USA Executive Director Frederick Isasi will step down from the organization at the end of the year.

Dr. Steven P. Furr, a family physician in Jackson, Ala., is now president of the American Academy of Family Physicians.

A message from Coalition for Affordable Prescription Drugs:

New polling shows that employers want options to choose the type of coverage that meets the unique needs of their employees. 89% of employers say their PBM is valuable in helping their organization offer affordable benefits to employees – and 91% of employers say it is important to have flexibility and choice in how their organization uses rebate savings. Read the survey results.

Document Drawer

The FDA warns consumers not to purchase or use 26 over-the-counter eye-drop products due to the potential risk of eye infections that could lead to partial vision loss or blindness.

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