Beremagene geperpavec

Abeona Therapeutics received FDA approval for its cell therapy to treat “butterfly skin,” a rare genetic disease that causes fragile skin and wounds. And its price tag makes it one of the most expensive therapies in the world. The cell therapy, known generically as pz-cel, will be marketed as Zevaskyn. It comes as credit card-sized skin sheets which are applied via surgery. It’s indicated for children and adults with recessive dystrophic epidermolysis bullosa, or RDEB, a skin disease that’s caused by a mutation in a gene for a type of collagen that keeps the skin layers together. The agency rejected the therapy last year over manufacturing questions. Abeona said Zevaskyn will cost $3.1 million, which puts its list price in range with other rare disease gene therapies. It is expected to be available in the third quarter of the year. The first therapy in the US to treat dystrophic epidermolysis bullosa was approved in 2023. Known as Vyjuvek, that treatment is sold by Krystal Biotech. It generated $290 million in revenue last year. That therapy costs about $631,000 per patient per year. Unlike Zevaskyn, Vyjuvek is administered once weekly. Both treatments have their roots in research conducted at Stanford, though Abeona says its therapy can potentially address more skin area than Vyjuvek can. Vyjuvek is a gene therapy that’s applied as a gel, while Zevaskyn is made from the patient’s own cells, which are harvested and then engineered to express the functional version of the collagen that people with DEB lack. In an interview, Abeona CEO Vishwas Seshadri said the company can manufacture 12 credit card-sized sheets at a time for each patient. Unlike typical cell and gene therapies, which are one-time treatments, the DEB treatments can be readministered. Abeona’s therapy was only administered once in clinical trial results reported so far. In a Phase 3 study, it reported that the cell therapy helped heal or shrink wounds in 11 children and adults. However, Seshadri said that in an ongoing study, patients were retreated with the skin sheets, though those data have not yet been shared publicly. In April 2024, the FDA issued a complete response letter to Abeona’s cell therapy. Seshadri said Abeona needed a faster test for sterility, which it then developed. The test is used to measure whether there are unwanted microorganisms, because its cell therapy has a short shelf life of just 84 hours. The company’s manufacturing site is in Cleveland. Along with the approval, Abeona gets a priority review voucher, which it could sell for over $100 million. As it stands right now, the priority review voucher program for rare pediatric disease is sunsetting, but rare disease advocates have pleaded with Congress to reauthorize the program, which they say provides an incentive, and in some cases a lifeline, for biotech companies to develop treatments for rare diseases.

The Food and Drug Administration on Tuesday approved a new treatment for a rare skin condition, teeing up a commercial battle between two cell and gene therapy developers.The agency cleared Abeona Therapeutics Zevaskyn for a form of epidermolysis bullosa, a rare disease that makes the skin susceptible to extensive and severe blistering. On a conference call, CEO Vishwas Seshadri disclosed the therapy a one-time treatment made from a persons skin cells will cost $3.1 million at list price, making it one of the worlds most expensive medicines.Abeona will reimburse insurers an unspecified percentage of the Zevaskyns cost if a treated wound needs additional therapy within three years, he said.There are several types of epidermolysis bullosa, or EB, an inherited condition that, according to some estimates, affects up to 50,000 people in the U.S. The most severe version is the recessive form of dystrophic disease, in which chronic blistering begins at birth or shortly afterwards and covers much of the skin, causing recurrent scarring, difficulty eating and other health problems.EB is typically managed through supportive care. However, in 2023, a topical gene therapy from Krystal Biotech was approved for people with the dystrophic form of the disease. That product, Vyjuvek, is administered weekly through a topical gel instead of a long-lasting skin graft and costs an estimated $631,000 per year. Its off to a strong sales start, making Krystal one of the few gene therapy developers to succeed commercially. Shares have more than doubled since Vyjuveks approval, giving Krystal a market value of nearly $5 billion.Abeona is positioning Zevaskyn as an alternative option, referring to it in a statement as the only FDA-approved product to treat disease-related wounds with a single application.The company is counting on the drug to help it break even financially next year. Zevaskyns approval also came with a special FDA voucher that Abeona can sell.Yet investors have been skeptical of Abeonas prospects because of the competition it faces from Krystals gene therapy. Its stock trades around $5 per share and fell 5% Tuesday morning following news of the approval.Some Wall Street analysts are optimistic Abeonas value will grow, though. In a report last year, analysts at the investment firm Stifel wrote Krystal and Abeonas therapies may be more complementary than competitive, since Vyjuvek likely isnt a one-size-fits-all treatment.Earlier this month, Jefferies analysts estimated Zevaskyn could generate $460 million in yearly sales at its peak, noting the clear unmet need Vyjuvek cannot fully address. Krystals treatment is a promising option for recurring small wounds, while Abeonas is more relevant for large, chronic wounds, they wrote.Abeonas Seshadri noted how the monthly retail cost of specialized wound care can reach $80,000, and the lifetime cost per patient for Vyjuvek can grow to tens of millions of dollars. Zevaskyns list price reflects its holistic value to patients, caregivers and the broader healthcare system, as it can treat small or large wounds with a single application, he said.Abeona will launch the treatment in the third quarter and expects gradual growth in uptake, with 10 to 15 patients anticipated to be treated this year and an acceleration afterwards, Seshadri said.The company hopes to sell that so-called priority review voucher, which expedites FDA drug reviews, in a reasonable timeframe. Abeona is prioritizing speed over price in selling it, as there is some uncertainty about the future of the FDA program governing these regulatory fast passes, Seshadri said.The FDA previously rejected Abeonas approval application last April due to questions over the drugs manufacturing. '