The most expensive drug ever approved by the FDA has — Sending UniQure Stock On A Warpath



A hemophilia B gene treatment developed by Carlisle Companies (CSL) and UniQure (QURE) has just received FDA approval; as a result, the price of QURE stock increased on Wednesday.

Shares of BioMarin (BMRN) surged as a result of the FDA's announcement that it would postpone a meeting of its advisory committee to consider its hemophilia A gene treatment. After considering the advantages and disadvantages of experimental medications, these advisers provide the FDA with non-binding recommendations. Roctavian from BioMarin has already received European approval.

The price of QURE stock increased 14.6% today, reaching 26.36. The stock's run after a 7.1% gain on Tuesday was prolonged as a result. MarketSmith.com reports that after consolidation, shares topped a purchase target at 25.91.

Stock in BioMarin increased 7.3% to 97.79. Due to this, BioMarin stock was able to enter a double-bottom base at 92.86 and break out of it.

Price Is Above Expectations for QURE Stock

Patients with hemophilia B who are undergoing Factor IX therapy or who have a history of potentially fatal hemorrhages or significant bleeding episodes are eligible to receive UniQure's infusion. Carlisle and UniQure are investing $3.5 million in the drug in the hopes that it will be a cure.

The clearance, according to analysts, is the best-case scenario for UniQure, the business that first developed Hemgenix before subsequently collaborating with Carlisle. There is no "black box" warning or restriction on the therapy's use to a particular group of patients on the label.

According to a note released on Wednesday by UBS analyst Eliana Merle, "given the uncertainties surrounding hemophilia gene therapy approvals and investor caution, we think this is a huge positive for UniQure and a crucial de-risking for the story."

The Institute for Clinical and Economic Review does not endorse the $3.5 million price tag. Most hemophilia gene treatments, according to the charity, would be financially viable at $2.5 million. According to the research, prices between $2 million and $3 million were investigated.

According to Joseph Schwartz, an analyst at SVB Securities, the smaller population of hemophilia B patients is probably the cause of the higher-than-expected pricing. He still sees the approval as a plus for patients who have a need for "burdensome prophylactic medication" and frequently have bleeding episodes.

The three hours following the Hemgenix injection will be spent monitoring the patients. They will then require three months of weekly monitoring to look for any potential liver problems. Schwartz points out that Factor IX preventive therapy is normally administered weekly.

Over the expected liberalization from factor treatment, bleeding, and joint injury for a very long period, he noted in a study, "We feel these monitoring needs make logical and will be seen as a worthy compromise."

On the shares of QURE, he has an outperform rating.

Other Gene Therapy Efforts by UniQure

The agreement specifies that Carlisle will be in charge of commercializing Hemgenix. In exchange, UniQure will get a royalty on sales in the midteens to low-20% range as well as further milestone payments totaling up to $1.5 billion. Additionally, UniQure possesses manufacturing rights.

According to Schwartz, "We view (the) approval as an important de-risking event for UniQure's possible future cash flow." "We also believe that this is encouraging for the prospective approval of BioMarin's Roctavian in hemophilia A."

The clearance probably gives UniQure's efforts in other gene therapy fields more credibility. Regarding the company's efforts on Huntington's disease, investors are likely to be more wary. In the UniQure research, three individuals out of fourteen reported severe adverse effects.

With the first information on functional (goals), the Phase 1 and Phase 2 update, which is still planned for the second quarter of 2023, "may be an instructive trigger for the program," noted Merle, the UBS analyst.

She upgraded her price target from 40 to 42 and has a buy rating on the QURE stock.

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