Rocket Pharmaceuticals has successfully sold its pediatric rare disease priority review voucher for $180 million, a move that the company plans to leverage in support of its pipeline, which is now primarily focused on a gene therapy in development for a rare hereditary condition that lacks FDA-approved treatment.<\/p>\n
The announcement of the voucher sale was made on Friday. The New Jersey-based gene therapy developer did not disclose the identity of the purchaser, only indicating through a regulatory filing that the buyer is \u201ca large pharmaceutical company.\u201d<\/p>\n
The FDA granted the voucher in March, in conjunction with the accelerated approval of Rocket\u2019s Kresladi, the first gene therapy for type 1 leukocyte adhesion deficiency (LAD-1), a hereditary immunodeficiency that can be life-threatening for infants. These vouchers can be applied to expedite the review of another eligible treatment for a rare disease, which reduces the standard review time from 10 months to just six months. However, these vouchers are transferable, and many recipients opt to sell them.<\/p>\n
The sale price of $180 million for Rocket’s voucher underscores the increasing demand for these expedited regulatory passes, which has driven their prices higher. Historically, vouchers have hovered around $100 million, but last year, the value reached approximately $150 million. Initially established by Congress, the program was set to expire at the end of 2024 but was renewed earlier this year, extending its validity until September 30, 2029.<\/p>\n
Following the approval of Kresladi, the most advanced program in Rocket’s pipeline is now RP-A501, a gene therapy that has reached a pivotal phase 2 study for Danon disease, a lysosomal storage disorder that results in the accumulation of toxic substances in cells, leading to issues in both cardiac and skeletal muscles. Cardiac complications can be fatal. Danon disease is caused by a deficiency in LAMP2B, a crucial protein for cardiac function. RP-A501 utilizes an adeno-associated virus to deliver a functioning version of the gene responsible for producing LAMP2B.<\/p>\n
Last year, the clinical trial of RP-A501 was temporarily suspended following the death of a patient during the phase 2 study. At the time, six Danon patients had received the therapy. An investigation determined that the death was likely related to a medication used in the preconditioning regimen that prepares patients for gene therapy.<\/p>\n
In August, the FDA authorized the resumption of the study with a lower dose range aligned with doses associated with efficacy from phase 1 trials. Additionally, treatment for the first three patients will be administered sequentially, with a four-week interval between each. In its most recent quarterly financial report, Rocket indicated that updates on the phase 2 study would follow the analysis of data from these three patients.<\/p>\n
At the end of the first quarter of this year, Rocket reported having $144.4 million in cash, which is expected to fund its operations for at least the next 12 months. With the completion of the priority review voucher sale, Rocket stated that its total capital of approximately $322.6 million should support operations until the second quarter of 2028.<\/p>\n","protected":false},"excerpt":{"rendered":"
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