Ultragenyx Pharmaceutical agrees to sell rare pediatric voucher for US$210M
The voucher came with Genglycos approval; payment awaits an antitrust waiting period and other closing conditions.
Adrian Kessler
A US$210 million payment is in prospect for Ultragenyx Pharmaceutical Inc. NASDAQRARE after it agreed on Wednesday to sell a rare pediatric disease priority review voucher earned from an FDA approval. The proposed price is nearly half the US$436 million it held in cash, cash equivalents and marketable securities at June 30.
The Oct. 7 announcement makes closing subject to customary conditions, including expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. Ultragenyx did not identify the buyer or give a closing date in that announcement.
Ultragenyx develops and sells treatments for rare genetic diseases from its California base. Its Genglycos gene therapy for glycogen storage disease type Ia brought the company the voucher when the FDA approved the treatment in August.
Shares rose 2.97 per cent to US$14.89 at the Nasdaq close on Oct. 7, according to the NASDAQRARE quote as of 4 p.m. EDT. The move followed Wednesday's announcement, which set no timetable for receiving the cash.
The Voucher Came With an August Approval
The FDA granted accelerated approval to Genglycos on Aug. 19 for patients aged eight and older, as an adjunct to nutritional management to reduce daily cornstarch intake. The approval was based on a reduction in cornstarch use, and the agency requires further trials to confirm clinical benefit.
The voucher itself is a separate asset from Genglycos. Under the FDA's rare pediatric disease program, its holder can use it to seek priority review for a different application or sell it to another sponsor. This agreement would transfer that option to the buyer once it closes.
“Monetizing this PRV provides significant non-dilutive capital to advance our efforts to bring forward first-ever therapies for rare and ultra-rare diseases, and supports our path to profitability,” Ultragenyx chief financial officer and executive vice-president of corporate strategy Howard Horn said in the Oct. 7 release.
Ultragenyx's Aug. 4 financial update also reported US$97 million in net cash used in operations during the second quarter of 2026. Its net loss for that quarter was US$92 million, compared with US$115 million a year earlier. The Oct. 7 release supplied no updated cash runway or estimate of the company's balance after the sale closes.
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Other Voucher Agreements Set a Price Range
Rocket Pharmaceuticals, Inc. NASDAQRCKT announced a US$180 million voucher agreement on April 28 after accelerated approval of its Kresladi gene therapy. Rocket said the proceeds would support its cardiovascular gene therapy pipeline and projected a cash runway into the second quarter of 2028.
Fortress Biotech, Inc. NASDAQFBIO said its majority-owned subsidiary Cyprium Therapeutics agreed on Feb. 23 to sell a voucher for US$205 million in gross proceeds. Cyprium said 20 per cent of those proceeds would go to a U.S. National Institutes of Health institute, so its gross sale price is not the amount it would keep.
The April and February agreements put Ultragenyx's US$210 million price in the range of recent voucher transactions. Unlike an equity offering, a voucher sale monetizes an FDA-awarded asset; Ultragenyx would give up the right to use or sell this voucher after closing.
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Ultragenyx projected fourth-quarter 2026 results from the dose-finding stage of its pivotal UX701 study in Wilson disease in its Aug. 4 update. Those data will test the doses under study, while the voucher proceeds still depend on satisfying the agreement's closing conditions.
