The FDA Just Approved Its Most Expensive Drug - Ever

The FDA Just Approved Its Most Expensive Drug – Ever

The Food and Drug Administration just approved its most expensive drug ever – a gene therapy for hemophilia B from Carlisle Enterprises (CSL) and Unique (QURE) – and QURE stock jumped on Wednesday.


Meanwhile, BioMarin (BMRN) erupted after the FDA said it would not hold an advisory committee meeting to discuss its hemophilia A gene therapy. These advisers make non-binding recommendations to the FDA after discussing the benefits and risks of experimental drugs. BioMarin’s Roctavian is already approved in Europe.

In today’s stock market, QURE stock jumped 14.6% to 26.36. That extended the title’s run after rising 7.1% on Tuesday. Shares rose above a buy point at 25.91 ex-consolidation, according to

BioMarin stock jumped 7.3% to 97.79. This pushed BioMarin stock out of a double-bottom base with an entry at 92.86.

QURE Stock: Price Above Expectations

UniQure infusion is approved for patients with hemophilia B who are currently on a preventive regimen called factor IX therapy, or who have a history of life-threatening bleeding or serious bleeding episodes. At a one-time cost of $3.5 million, Carlisle and UniQure hope the drug is a cure.

Analysts say the approval is the best-case scenario for UniQure, the company that originally developed Hemgenix before partnering with Carlisle in later stages. The label does not limit the therapy to a specific group of patients and does not include a “black box” warning.

“Given the uncertainty surrounding hemophilia gene therapy approvals and cautious investors, we believe this is a major positive for UniQure and a key de-risking story,” said UBS analyst Eliana Merle in a report Wednesday.

The $3.5 million price tag is above a recommendation from the Institute for Clinical and Economic Review. The nonprofit said most gene therapies for hemophilia would pay off at $2.5 million. The report looked at prices between $2 million and $3 million based on executive feedback.

SVB Securities analyst Joseph Schwartz said the higher-than-expected price likely explains the small patient population with hemophilia B. Still, he views the approval as positive for patients with frequent bleeding episodes and the need for “tedious prophylactic treatment”.

Patients will be monitored for three hours after the Hemgenix infusion. Then they will need to be monitored weekly for three months to monitor for potential liver complications. Schwartz notes that preventive treatment with factor IX is usually given weekly.

“We believe these monitoring requirements make sense and will be seen as a worthwhile sacrifice for the likely liberalization of factor therapy, bleeding and joint injury for a long time to come,” he said in a statement.

It has an outperform rating on QURE shares.

Other UniQre Gene Therapy Efforts

Under the terms of the agreement, Carlisle will be responsible for commercializing Hemgenix. In return, UniQure will receive up to $1.5 billion in additional milestone payments and a 12-20% sales royalty. UniQure also has manufacturing rights.

“We view (the) approval as a key de-risking event for UniQure’s future cash flow potential,” Schwartz said. “We also believe this bodes well for the approval potential of BioMarin’s Roctavian in hemophilia A.”

The approval also likely lends credence to UniQure’s efforts in other areas of gene therapy. Investors are probably more cautious about the company’s work in Huntington’s disease. Three of the 14 patients in the UniQure study experienced serious side effects.

“We note that the Phase 1 and Phase 2 update is still guided for the second quarter of 2023 and could be an informative catalyst for the program with early data on functional (targets),” Merle said. , the UBS analyst.

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

Follow Allison Gatlin on Twitter at @IBD_AGatlin.


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