$2mn In The US, Gene Therapies Might Become The Standard

In recent years, the high cost of gene therapies has emerged as a major barrier to the development of personalised medicines. Most notably, bluebird Bio was forced to pull its blood disorder medicine Zynteglo from the European market after encountering an unworkable pricing situation in the region.

However, the one-time beta-thalassemia therapy price charged by bluebird in the EU, which was initially offered at $1.8 million in June 2019, may start to be accepted in the United States. That’s the low end, too.

A report on the clinical efficacy and economics of CSL Behring’s etranacogene dezaparvovec for haemophilia B was released by the Institute for Clinical and Economic Review (ICER) on November 2. The U.S. drug cost watchdog also revised its prior assessment of BioMarin’s valoctocogene roxaparvovec, which recently won approval in Europe as Roctavian, for haemophilia A.

According to the ICER evaluation, CSL’s medication, which is scheduled for an FDA priority review decision at the end of November, would be fairly priced at around $2.9 million. The business resubmitted BioMarin’s Roctavian to the U.S. FDA in late September at a price of about $1.9 million, which would be acceptable. ICER warned in a release that duration of effectiveness with gene treatments and risks for uncommon side events remain important concerns.

Before a virtual public meeting on November 18, where an ICER committee will assess the evidence on the medications and hear additional testimony, the watchdog’s review will be conducted. The specialists will also discuss the treatments’ relative efficacy as well as their additional advantages and long-term worth.

ICER, however, mostly supported the high million-dollar price stickers for both BioMarin’s and CSL’s medications in its own study. Due to the absence of the necessity for relatively expensive preventative treatment, both medicines exhibit health improvements and the possibility of significant cost offsets. Roctavian’s net price in Europe is approximately 1.5 million euros (or $1.46 million), according to BioMarin this summer. The drug would still be sensibly valued at up to $1.9 million, according to ICER’s most recent estimate, although BioMarin might go as high as $2.5 million.

According to BioMarin, the company anticipates making its first commercial Roctavian sales in Europe in the fourth quarter. The U.S. FDA authorised bluebird’s $2.8 million medication Zynteglo in August, and the firm thinks the high price reflects the benefit of a one-time treatment in the U.S., according to the company’s chief commercial officer, Tom Klima, in an interview earlier this year.

In addition, shortly after Zynteglo received permission in the United States, bluebird in September successfully obtained approval for eli-cel, which is now known as Skysona, for the extremely uncommon neurological condition cerebral adrenoleukodystrophy (CALD). While there is little doubt that gene treatments are transforming the landscape of cancer and rare illness treatment, Brett Sahli, Prime Therapeutics’ assistant vice president of pharmaceutical trade affairs, warned that there is always a risk of cost shocks.

Pricing for therapies in the millions of dollars makes access impossible in many places, Pearson said, unless it’s on a totally compassionate basis or another basis outside the developed countries. The dilemma is even greater globally, ICER president Steven Pearson, M.D., M.Sc., said on the panel, noting gene therapy is still a fairly dicey business proposition for companies in the field.