The FDA has just approved its most expensive drug - ever

The Food and Drug Administration has just approved its most expensive drug yet, a gene therapy for hemophilia B from Carlisle Companies (CSL) and UniQure (QURE) — and QURE shares soared on Wednesday.


Meanwhile, BioMarin (BMRN) shares soared after the FDA said it would not hold an advisory committee meeting to discuss its gene therapy for hemophilia A. Those advisers make nonbinding recommendations to the FDA after discussing the benefits and risks of experimental drugs. BioMarin’s Roctavian is now approved in Europe.

On today’s stock market, QURE shares jumped 14.6% to 26.36. That extended the stock’s run from Tuesday’s 7.1% jump. Stocks topped a point of purchase at 25.91 out of consolidation, acc

BioMarin shares jumped 7.3% to 97.79. That sent BioMarin shares out of a double bottom base with input at 92.86.

QURE Stock: The price is above expectations

The UniQure infusion is approved for patients with hemophilia B who are currently on a prevention regimen called factor IX therapy, or who have a history of potentially fatal 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 approval is the best-case scenario for UniQure, the company that originally developed Hemgenix before partnering with Carlisle in the 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 investor caution, we think this is a big positive for UniQure and a key de-risk for the story,” UBS analyst Eliana Merle said in a report on Wednesday.

The $3.5 million price tag is above the Institute for Clinical and Economic Review’s recommendation. The nonprofit said the most gene therapies for hemophilia would be profitable at $2.5 million. The report looked at prices between $2 million and $3 million based on comments from executives.

SVB Securities analyst Joseph Schwartz said the higher-than-expected price was likely due to the small population of hemophilia B patients. Still, he saw the approval as positive for patients with frequent bleeding episodes and the need for “burdensome prophylactic therapy.”

Patients will be monitored for three hours after the Hemgenix infusion. They will then have to undergo weekly monitoring for three months to watch 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 damage over the long term,” he said in a report.

He has an outperform rating on QURE shares.

UniQure’s other gene therapy efforts

Under the terms of the agreement, Carlisle will be responsible for the commercialization of Hemgenix. In return, UniQure will receive up to $1.5 billion in additional milestone payments and a sales royalty in the mid to low 20% range. UniQure also has production rights.

“We see (this) approval as a key de-risking event for UniQure’s future cash flow potential,” Schwartz said. “We also think this bodes well for the potential for approval 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 likely more cautious about the company’s work in Huntington’s disease. Three of 14 patients in the UniQure study had serious side effects.

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

She has a buy rating on QURE shares and raised her target price to 42 out of 40.

Follow Alison Gatlin on Twitter at @IBD_AGatlin.


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