Despite competitive pressure from a buzzy CRISPR therapy, bluebird bio keeps striding forward with its sickle cell disease gene therapy Lyfgenia.
The Massachusetts biotech has signed a second outcomes-based reimbursement agreement for Lyfgenia, according to a securities filing. The deal pushes Lyfgenia’s coverage range to nearly 200 million people in the U.S., up from the previous 100 million when bluebird signed the first agreement three weeks ago.
Bluebird pulled off the feat within a month of winning FDA approval for Lyfgenia in SCD patients who have a history of the painful blood flow blockade episodes known as vaso-occlusive events. And it comes despite the company pricing Lyfgenia at a wholesale acquisition cost that’s about 40% higher than Vertex and CRISPR Therapeutics’ rival gene therapy Casgevy.
Under these outcomes-based agreements, bluebird is offering a discount if a patient is hospitalized because of vaso-occlusion events within three years after treatment. In addition to commercial payers, bluebird has said it’s offering contracting options to state Medicaid agencies as well.
The two therapies have shown similar efficacy in keeping vaso-occlusion at bay. But analysts hold different opinions about which company may be more successful when it comes to their marketing promotion efforts.
Although the FDA approved Lyfgenia and Casgevy at the same time, bluebird got a head start on its launch preparations. Bluebird had already been selling Zynteglo, a twin med to Lyfgenia for beta thalassemia, since fall 2022, and it has built a network of qualified treatment centers, which Casgevy can’t match at the moment.
But a William Blair team previously pointed to Vertex’s “extensive commercial experience” built on a successful cystic fibrosis franchise as a threat to bluebird.
Following Lyfgenia’s approval and a surprise lack of an FDA priority review voucher, bluebird in December launched a $150 million public offering to support commercialization. Although the dilutive capital raise could alleviate a near-term cash constraint, “the pathway to cash flow breakeven is still murky in absence of truly remarkable launch acceleration,” Leerink Partners analysts wrote in a December note.
Lyfgenia’s black-box warning and higher list price could be challenges in its competition with Casgevy, the Leerink team said.
Bluebird will provide an update on Lyfgenia’s launch progress at the upcoming J.P. Morgan Healthcare Conference on Tuesday.
PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.
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