The U.S. Food and Drug Administration approved on Monday PTC Therapeutics, Inc.’s (NASDAQ:PTCT) SEPHIENCE (sepiapterin) for children and adult patients with phenylketonuria (PKU).
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PKU is a rare, inherited disorder that prevents the body from breaking down phenylalanine, an amino acid in protein-rich foods. PKU can lead to intellectual disability, seizures and behavioral problems.
The approval includes broad labeling for hyperphenylalaninemia (HPA) in adult and pediatric patients one month of age and older with sepiapterin-responsive PKU.
HPA is the most common amino acid metabolism defect in humans. It is characterized by elevated levels of the amino acid phenylalanine in the blood.
“We are excited to have reached this important milestone for those affected by PKU,” said Matthew B. Klein, CEO of PTC Therapeutics. “The Sephience clinical data along with our expertise in launching rare disease therapies position Sephience to become the future standard of care,” Klein added.
The FDA approval is based on the evidence of significant efficacy and safety from the Phase 3 APHENITY trial and the durability of the treatment effect in the APHENITY long-term extension study.
William Blair writes that, given the approximately 17,000 PKU patients in the U.S., management announced its initial commercial launch strategy will target 1,200 prescribers across 104 centers, accounting for over 80% of PKU claims data.
Sephience’s wholesale acquisition cost (WAC) is approximately $40,000 a month or $480,000 per year.
In addition, the company will initially prioritize patients who have failed or are not well controlled on oral therapies, such as Biomarin Pharmaceutical Inc.’s (NASDAQ:BMRN) Kuvan (sapropterin dihydrochloride), which it estimates to include 40% of the PKU population.
While the company will not specifically target treatment-naïve patients initially, management stated it has seen interest from some treatment-naïve patients in trying Sephience.
Management expects start forms to be available immediately, with the first shipments to U.S. patients to begin in August.
In its investor presentation, PTC expects an over $1 billion potential revenue opportunity for Sephience.
William Blair views the approval of Sephience in the U.S. with a broad label as a significant achievement for PTC and says it could become the new standard of care for PKU patients.
Analyst Sami Corwin sees Sephience as the company’s key revenue driver in the future and writes that it will be a crucial asset in aiding PTC in achieving cash flow positivity.
William Blair maintains the Outperform rating and models Sephience’s peak global sales of $2.2 billion in 2031 and a 2025 total revenue of $1.71 billion.
Other Analyst Ratings:
UBS maintains PTC Therapeutics with a Buy, raising the price forecast from $71 to $80.
Cantor Fitzgerald maintains PTC Therapeutics with an Overweight, raising the price forecast from $112 to $120.
Wells Fargo maintains PTC Therapeutics with an Overweight, raising the price forecast from $74 to $78.
Barclays maintains PTC Therapeutics with an Equal-Weight, raising the price forecast from $42 to $46.
PTCT Price Action: PTC stock is up 12.44% at $49.86 at publication on Tuesday.
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