FOIAengine: Sofinnova Bet on Drug FDA Approved Last Week
Shares of Ultragenyx Pharmaceutical Inc. rose 16 percent September 17 after the Food and Drug Administration approved Fayuvi, its gene therapy for Sanfilippo syndrome Type A, a rare and devastating genetic disease that strikes children and progressively damages the brain and nervous system. Investment firm Sofinnova Investments, holder of a significant stake in Ultragenyx, was on the alert for Thursday’s FDA decision. Ahead of that, the investment firm filed a July 28 Freedom of Information Act request with the FDA about Ultragenyx’s manufacturing compliance.
Sofinnova is a fifty-year-old healthcare investment firm with approximately $4.6 billion in assets under management, primarily focused on therapeutic drugs. According to a June Securities and Exchange Commission 13F filing (the most recent available), Sofinnova was not merely watching from the sidelines. A month before it filed its July request, the firm held more than 1.7 million shares of Ultragenyx worth about $57 million, along with call options covering another $16.7 million in shares.
Sofinnova was a large shareholder at the same time an unusually large and rising portion of Ultragenyx’s tradable shares were being sold short. Although not indicative of Sofinnova’s investment strategy, the significant short interest indicates that the market was sharply divided about Ultragenyx during the period surrounding the FDA decision.
Followers of FOIAengine, which attracts requests in as close to real time as available, already know how investment professionals use FOIA requests as an important tool in analyzing the ups and downs of a drug’s lengthy regulatory and market history. In the case of Fayuvi, an important part of that history was the FDA’s investigation of issues at the Ultragenyx manufacturing facility in Bedford, Massachusetts. In April 2025, FDA inspectors issued a seven-page Form 483 detailing serious manufacturing and safety violations. Form 483 is the document the agency gives a company when inspectors identify conditions that may violate federal requirements.
The safety issues had not been resolved by July 2025, when the FDA rejected Ultragenyx’s initial application for Fayuvi, which was then known as UX111. The rejection was based on manufacturing-control issues and observations from inspections of the Bedford plant and a third-party manufacturer. The company issued a press release saying it was “working with urgency to respond and resubmit.” This was critical to their prospects, because Ultragenyx had identified Bedford as one of the two facilities that would manufacture Fayuvi.
In January of this year Ultragenyx resubmitted the application, saying it had addressed the manufacturing issues, but the FDA initially asked for additional supporting documentation. Finally, in April the FDA accepted the resubmission and set last Thursday, September 19, as the Fayuvi decision date.
From April 6 to 14 the FDA conducted yet another inspection at Bedford and issued another Form 483. An FDA regulatory review resolved the concerns, and the inspection was classified Voluntary Action Indicated.
During this process Fayuvi’s clinical record was strong, so investors knew that the primary risk facing the application was possible FDA concern about safety issues at the Bedford facility.
With millions at stake, on July 26 Seth Filbrun, a Public Equity Associate at Sofinnova, filed a FOIA request with the FDA seeking “any Form 483 that has been issued for the following manufacturing facility this year. Ultragenyx Pharmaceutical Inc., 170 Middlesex Turnpike, Bedford, Massachusetts 01730.”
Sofinnova apparently was seeking to resolve one of the most important remaining uncertainties surrounding the drug’s approval. And the news turned out to be positive.
FOIA Roundup: In other developments, a review of all FDA FOIA requests submitted by financial firms during July and August found 31 requests from investment firms, banks and other financial organizations. Eighteen sought Form 483s, inspection reports, inspection classifications, manufacturing records, or closely related documents.
The logs suggest that, like Sofinnova, investors are using the requests to resolve specific uncertainties that remain after companies have made their public disclosures.
For example: after Indian drugmaker Dr. Reddy’s Laboratories disclosed that an inspection of its biologics plant had resulted in seven observations, four different financial organizations — Spark Institutional Equities, Nomura, Jefferies India and BofA Securities — filed requests for essentially the same FDA inspection document. Nomura soon requested a Form 483 from another Dr. Reddy’s plant that had just been inspected.
Point72 made three requests in August involving Filspari, a kidney-disease drug, seeking detailed records for ten specifically identified adverse-event cases. The precision is notable. Point72 staff already had examined the public adverse-event database, identified cases of interest, and were seeking the underlying reports to learn more about the cases.
Jefferies asked for a specific statistic involving Spravato, Johnson & Johnson’s treatment for depression: the number of health care settings certified to administer the drug. Because Spravato can be provided only through specially certified locations, growth in those locations can offer a window into the infrastructure available to generate future sales
And OrbiMed asked for the minutes from FDA meetings with uniQure concerning AMT-130, an experimental treatment for Huntington’s disease, after public accounts of the agency’s regulatory position had changed. The apparent question was: What exactly had the FDA told the company?
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Randy E. Miller, co-creator of FOIAengine, is a Washington lawyer, publisher, and former government official. He has developed several online information products and was a partner at Hogan Lovells, where he founded the firm’s Brussels office and represented clients on international regulatory matters. Miller also has served as a White House trade lawyer, Senior Legal Adviser to the U.S. Mission to the World Trade Organization, policy director to Senator Bob Dole, and adjunct professor at Georgetown University. He is a graduate of Yale and Georgetown Law. FOIAengine is a product of PoliScio Analytics (PoliScio.com), a venture specializing in U.S. political and governmental research, co-founded by Miller and Washington journalist John A. Jenkins.
Write to Randy E. Miller at randy@poliscio.com.