ADDED
As of February 18, 2026, the registrant had 82,774,730 shares of Common Stock, $0.001 par value per share, outstanding.
Summary of Risk Factors We may be unable to continue to execute our commercial strategy for our products, fail to obtain renewal of, or satisfy the conditions of our marketing authorization for our products; Delays or failures in obtaining regulatory approval would materially impair our commercialization capabilities; We may be unable to continue to commercialize Translarna for nmDMD in the EEA if individual countries in the EU do not leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued commercial use of Translarna.
Business Overview We are a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders.
We are advancing a robust and diversified pipeline of transformative medicines as part of our mission to provide access to best-in-class treatments for patients with unmet medical needs.
Our strategy is to leverage our scientific expertise and global commercial infrastructure to optimize value for our patients and other stakeholders.
The disclosure below summarizes the status of our significant commercial products and clinical-stage programs as of the date of this report, including those with our strategic partners: Global Commercial Footprint o Sephience TM (sepiapterin) Sephience is our product for the treatment of adult and pediatric patients with phenylketonuria, or PKU, a rare, inherited metabolic disease, characterized by the body's inability to break down an essential amino acid called phenylalanine, or Phe, which can result in neurological and other symptoms.
Through its novel dual mechanism of action, Sephience is able to effectively reduce blood Phe levels and has the potential to treat a broad range of PKU patients.
Sephience is approved in the United States, European Economic Area, or EEA, Japan and additional geographies.
o Global DMD Franchise We have two products, Translarna TM (ataluren) and Emflaza (deflazacort), for the treatment of Duchenne muscular dystrophy, or DMD, a rare, life-threatening disorder.
While Translarna previously had conditional approval in the EEA, in March 2025, the European Commission, or EC, adopted the negative opinion of the Committee of Medicinal Products for Human Use, or CHMP, of the European Medicines Agency, or EMA, to not renew the conditional marketing authorization of Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy, or nmDMD.
REMOVED
As of February 25, 2025, the registrant had 78,869,368 shares of Common Stock, $0.001 par value per share, outstanding.
Summary of Risk Factors We may be unable to continue to execute our commercial strategy for our products, fail to obtain renewal of, or satisfy the conditions of our marketing authorization for our products; Delays or failures in obtaining regulatory approval would materially impair our commercialization capabilities; We may be unable to continue to commercialize Translarna for nmDMD in the EEA if the EC adopts the negative opinion issued by the CHMP for the renewal of the existing conditional authorization for Translarna.
Business Overview We are a global biopharmaceutical company that discovers, develops and commercializes clinically differentiated medicines that provide benefits to children and adults living with rare disorders.
Our ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines.
Our mission is to provide access to best-in-class treatments for patients who have little to no treatment options.
Our strategy is to leverage our strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients.
We believe that this allows us to maximize value for all of our stakeholders.
The disclosure below summarizes the status of our significant clinical-stage programs and commercial products as of the date of this report, including those with our strategic partners: Global Commercial Footprint o Global DMD Franchise We have two products, Translarna TM (ataluren) and Emflaza (deflazacort), for the treatment of Duchenne muscular dystrophy, or DMD, a rare, life-threatening disorder.
Translarna currently has conditional marketing authorization in the European Economic Area, or EEA, for the treatment of nonsense mutation Duchenne muscular dystrophy, or nmDMD, in ambulatory patients aged two years and older.
In January 2024, the Committee of Medicinal Products for Human Use, or CHMP, of the European Medicines Agency, or EMA, issued a negative opinion for the renewal of the conditional marketing authorization following a re-examination procedure.