Rocket Pharmaceuticals sold its rare disease pediatric priority review voucher for $180 million, cash that the company will use to support a pipeline now led by a gene therapy in development for a rare, inherited disease with no FDA-approved therapies.
Rocket announced the voucher sale on Friday. The Cranbury, New Jersey-based gene therapy developer did not disclose who purchased the voucher, saying only in a regulatory filing that the buyer was “a large pharmaceutical company.”
The FDA awarded the voucher in March alongside the accelerated approval of Rocket’s Kresladi, the first gene therapy for leukocyte-adhesion deficiency type 1 (LAD-1), an inherited immunodeficiency that can become fatal to babies. Such vouchers may be applied to another eligible rare disease therapy, cutting the standard 10-month review down to six months. But vouchers are transferable and many recipients choose to sell them.
The $180 million price for Rocket’s voucher shows that growing demand for these regulatory fast passes is raising their prices. For many years, voucher fetched prices of around $100 million. Last year, voucher prices reached around $150 million. The Congressionally created program lapsed at the end of 2024 but was renewed earlier this year. The program has been extended to Sept. 30, 2029.
After Kresladi’s approval, the most advanced program in the Rocket pipeline is now RP-A501, a gene therapy that has reached a pivotal Phase 2 study in Danon disease, a lysosomal storage disorder that leads to the buildup of toxic substances in cells that manifest as problems in cardiac and skeletal muscle. The heart problems can become fatal. Danon is caused by deficiency of LAMP2B, a protein that’s important to cardiac function. RP-A501 uses an adeno-associated virus to deliver a functioning version of the gene that produces the instructions for making LAMP2B.
Last year, a patient death in RP-A501’s Phase 2 study led to an FDA clinical hold on the trial. At the time of the clinical hold, six Danon patients had received RP-A501. An investigation found the death was likely due to a drug used in the preconditioning regimen that prepares a patient for the gene therapy.
Last August, the FDA permitted resumption of the study with a lower range of doses that aligns with doses associated with efficacy in Phase 1 testing. Also, treatment of the first three patients will be done sequentially with a four-week interval between them. In its most recent quarterly financial report, Rocket said it would provide updates on the Phase 2 study following the review of data from these next three patients.
As of the end of the first quarter of this year, Rocket reported its cash position of $144.4 million would fund operations for at least the next 12 months. With the priority review voucher sale now complete, Rocket said its capital totaling about $322.6 million is expected to fund operations into the second quarter of 2028.
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