Sanfilippo Gene Therapy Approved at $4 Million; BMS Ends Aurum Drug Development

The US Food and Drug Administration (FDA) approved the first gene therapy for the rare neurologic disease Sanfilippo syndrome type A, while Bristol Myers Squibb (BMS) discontinued development of an oncology drug licensed from Aurum Therapeutics.

According to BioPharmaDive on the 18th (local time), the FDA approved Pavyu, a treatment for Sanfilippo syndrome type A. The price was set at $3.95 million (approximately 530 million won).

Pavyu is the second gene therapy Ultragenyx Pharmaceutical has brought to market since August. Neither therapy is expected to generate blockbuster-level sales, but with the approval, Ultragenyx secured a priority review voucher that can be sold.

William Blair analyst Sami Corwin said the voucher would "significantly strengthen" the company's financial statements. Ultragenyx had signaled that it would pursue cost reductions after its higher-revenue-potential treatment for Angelman syndrome failed. The company's shares posted a double-digit gain after Pavyu's approval.

BMS has discontinued development of an experimental oncology drug it licensed from startup Aurum Therapeutics three years ago. In 2023, BMS paid Aurum a $100 million upfront payment (approximately 134 billion won) to secure the rights to ORM-6151.

The treatment combines an antibody-drug conjugate (ADC) with protein degradation technology and was tested in acute myeloid leukemia. Aurum said in regulatory filings that it decided to end the drug's development after reviewing Phase 1 trial data. Aurum was eligible to receive up to $80 million (approximately 107.2 billion won) in contingent payments under the deal.

Roche's blood cancer treatment Lunsumio succeeded in a late-stage trial in follicular lymphoma. Roche did not disclose specific figures but said the regimen combining Lunsumio with standard therapy significantly improved progression-free survival compared with Rituxan and existing treatment.

Lunsumio is currently approved as a third-line treatment, and the Phase 3 results could lead to approval in standard treatment. Roche plans to submit the results to regulators.

Longeveron will begin a strategic review after its cardiac disease cell therapy laromestrocel failed in a Phase 2 trial. After 12 weeks of follow-up, there was no statistically significant change in a measure of the heart's blood-pumping capacity.

Longeveron plans to discuss next steps with the FDA, hire an investment bank to "review all options" and reduce costs.

Rocket Pharmaceuticals reached an agreement with US regulators on proposed changes to the pivotal trial of its treatment for the rare cardiac disease Danon disease. Rocket previously paused the trial after one participant died, then modified and resumed the protocol; safety was subsequently confirmed in three participants.

Rocket said on the 17th that the FDA had approved a safety update and a path to completing the trial. The company plans to enroll nine additional patients and complete dosing by mid-2027, and will disclose the program's progress at an investor webinar in early October.