Replies to post #177054 on Biotech Values

[DewDiligence]

QURE—…the hemophilia B gene therapy could be a game changer as it offers the potential to eliminate the need for routine Factor IX replacement.

The first time I read about the concept of gene therapy many years ago, the application being discussed was hemophilia. It’s been a long time coming.

From a business standpoint, is there a bona fide need for a (presumably very expensive) gene therapy for hemophilia B now that there are multiple long-acting FIX products on the market or nearing commercialization?

[DewDiligence]

QURE gene therapy breaks the $1M price barrier:

http://in.reuters.com/article/2014/11/26/us-health-genetherapy-price-idINKCN0JA1TP20141126

The Western world's first gene therapy drug is set to go on sale in Germany with a 1.1 million euro ($1.4 million) price tag, a new record for a medicine to treat a rare disease. The sky-high cost of Glybera, from Dutch biotech firm UniQure and its unlisted Italian marketing partner Chiesi, shows how single curative therapies to fix faulty genes may upend the conventional pharmaceutical business model.

…The new drug fights an ultra-rare genetic disease called lipoprotein lipase deficiency (LPLD) that clogs the blood with fat. The medicine was approved in Europe two years ago but its launch was delayed to allow for the collection of six-year follow-up data on its benefits.

…Chiesi…is seeking a retail price of 53,000 euros per vial, or 43,870 euros ex-factory [i.e. wholesale]. That equates to 1.11 million euros for a typical LPLD patient, averaging 62.5 kg in clinical trials, who will need 42 injections from 21 vials.