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.

周二，食品和药物管理局批准了一种用于治疗罕见皮肤病的新疗法，这将引发两家细胞和基因疗法开发商之间的商业竞争。

The agency

机构

cleared Abeona Therapeutics’ Zevaskyn

清除了Abeona Therapeutics的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 person’s skin cells — will cost $3.1 million at list price, making it one of the world’s most expensive medicines..

一种罕见的表皮松解症，这种疾病使皮肤容易出现广泛且严重的水泡。在一次电话会议上，首席执行官 Vishwas Seshadri 披露了这种疗法——一种由患者皮肤细胞制成的一次性治疗——其定价为 310 万美元，成为全球最昂贵的药物之一。

Abeona will reimburse insurers an unspecified percentage of the Zevaskyn’s cost if a treated wound needs additional therapy within three years, he said.

他说，如果治疗后的伤口在三年内需要额外的治疗，Abeona公司将向保险公司偿还Zevaskyn的一部分费用，具体百分比未定。

There are several types of epidermolysis bullosa, or EB, an inherited condition that, according to some estimates, affects up to

大疱性表皮松解症（简称EB）是一种遗传病，据一些估计，可能影响多达

50,000 people in the U.S.

美国有 5 万人

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.

最严重的形式是“营养不良性”疾病的隐性形式，这种疾病从出生时或出生后不久开始出现慢性水疱，并覆盖大部分皮肤，导致反复结疤、进食困难和其他健康问题。

EB is typically managed through supportive care. However, in 2023, a

EB通常通过支持性护理进行管理。然而，在2023年，

topical gene therapy from Krystal Biotech

来自Krystal生物技术的局部基因治疗

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

适用于患有该病营养不良型的患者。该产品名为Vyjuvek，通过每周使用局部凝胶给药，而非持久性皮肤移植，并且费用为

estimated $631,000 per year

估计每年631,000美元

. It’s off to a

. 它开始了

strong sales start

强劲的销售开端

, making Krystal one of the few gene therapy developers to succeed commercially. Shares have more than doubled since Vyjuvek’s approval, giving Krystal a market value of nearly $5 billion.

，这使得Krystal成为少数几家在商业上取得成功的基因疗法开发商之一。自Vyjuvek获批以来，其股价已经翻了一倍多，使Krystal的市值接近50亿美元。

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.”

阿贝奥纳公司正在将Zevaskyn定位为一种替代选择，并在一份声明中称其为唯一获得FDA批准的、通过“单次应用”治疗疾病相关伤口的产品。

The company is counting on the drug to help it break even financially next year. Zevaskyn’s approval also came with a special FDA voucher that Abeona can sell.

公司指望该药物能帮助公司在明年实现财务上的收支平衡。Zevaskyn的批准还附带了一张Abeona可以出售的特殊FDA券。

Yet investors have been skeptical of Abeona’s prospects because of the competition it faces from Krystal’s gene therapy. Its stock trades around $5 per share and fell 5% Tuesday morning following news of the approval.

然而，由于面临来自 Krystal 基因疗法的竞争，投资者对 Abeona 的前景持怀疑态度。其股票每股交易价格约为 5 美元，并在周二早间批准消息传出后下跌了 5%。

Some Wall Street analysts are optimistic Abeona’s value will grow, though. In a report last year, analysts at the investment firm Stifel wrote Krystal and Abeona’s therapies may be “more complementary than competitive,” since Vyjuvek likely isn’t a “one-size-fits-all” treatment.

一些华尔街分析师对 Abeona 的价值增长持乐观态度。然而，在去年的一份报告中，投资公司 Stifel 的分析师写道，Krystal 和 Abeona 的疗法可能“更具互补性而非竞争性”，因为 Vyjuvek 可能并不是一种“一刀切”的治疗方法。

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.” Krystal’s treatment is a “promising option for recurring small wounds,” while Abeona’s is “more relevant for large, chronic wounds,” they wrote..

本月早些时候，Jefferies分析师估计Zevaskyn在销售高峰期可能实现每年4.6亿美元的收入，指出Vyjuvek“无法完全满足的明确未满足需求”。他们写道，Krystal的治疗方法是“针对反复出现的小伤口的一种有前景的选择”，而Abeona的疗法则“更适用于大型慢性伤口”。

Abeona’s 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.” Zevaskyn’s 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公司的Seshadri指出，专门的伤口护理每月零售成本可达8万美元，而Vyjuvek每名患者的终身成本可能增长到“数千万美元”。他表示，Zevaskyn的定价反映了其对患者、护理人员以及更广泛的医疗保健系统的“整体价值”，因为它可以单次应用治疗小或大的伤口。

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.

Seshadri 表示，Abeona 将在第三季度推出该疗法，并预计接受治疗的患者人数将逐步增长，今年预计有 10 到 15 名患者接受治疗，之后将会“加速”。

The company hopes to sell taht so-called priority review voucher, which expedites FDA drug reviews, in a “reasonable timeframe.” The company 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..

公司希望在“合理的时间框架内”出售所谓的优先审查券，该券可加快 FDA 药物审查速度。Seshadri 表示，由于监管这些快速通道的 FDA 项目未来存在一些不确定性，公司在出售时将速度置于价格之上。

The FDA previously

FDA 之前

rejected Abeona’s approval application last April

去年四月拒绝了Abeona的批准申请

due to questions over the drug’s manufacturing.

由于对这种药物的生产存在疑问。