Prescription medicine
Sepiapterin Brand names: Sephience
Sepiapterin (Sephience) is a prescription medicine with an FDA approval on record since 2025. No generic is listed in FDA's NDC Directory.
- No generic listed
Check it at the source: the sepiapterin FDA label on DailyMed (effective April 24, 2026, PTC Therapeutics Inc.); the NDA 219666 record on Drugs@FDA; every sepiapterin label on DailyMed.
Highlights of the record
FDA label of April 24, 2026; NDC Directory, Drugs@FDA and Orange Book, October 8, 2026
Used for
From the label: indications and usageSEPHIENCE is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU).
Forms and strengths
- 250 mg
- 1000 mg
Powder.
On the market
- Earliest approval on file
- July 28, 2025 Sephience, Ptc Therap; Drugs@FDA’s file, where older or withdrawn approvals can be missing
- Generic approvals
- No generic approved
- Labelers listed (NDC)
- 2 of which 0 under generic applications, 2 under brand applications, repackagers included
- Last listed patent
- March 6, 2042 Sephience powder; not a generic launch date
Supply and safety
- Pharmacy cost (NADAC)
- Not in the survey
- Shortage (FDA)
- No current entry
- Recalls by its makers (FDA)
- No recall recorded
- FAERS reports
- 52 through July 30, 2026
What is sepiapterin used for?
From the label: indications and usageSEPHIENCE is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). SEPHIENCE is to be used in conjunction with a phenylalanine (Phe)-restricted diet. SEPHIENCE is a phenylalanine hydroxylase (PAH) activator indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). SEPHIENCE is to be used in conjunction with a phenylalanine (Phe)- restricted diet.
Source: FDA drug label (Sephience), PTC Therapeutics Inc., effective April 24, 2026 (SPL set 32ac719c-49f0-4105-9c46-18c19583a5c2), via openFDA; the full label on DailyMed
Is there a generic for sepiapterin (Sephience)?
No generic sepiapterin is approved (Drugs@FDA, October 8, 2026).
The Orange Book lists 4 patents for Sephience powder: the compound patent runs to April 16, 2038, and the last listed entry to March 6, 2042. A patent or exclusivity date is not the date a generic goes on sale: court cases and settlements move it either way.
- 2025 earliest approval on file
- 2042 last listed patent
Source: Drugs@FDA, FDA NDC Directory and the Orange Book (openFDA bulk files, October 8, 2026)
What does the sepiapterin label say about other medicines?
The sepiapterin label names 9 other medicines in its interaction, contraindication or warning sections. The sentences are the labels' own; a label lists what its studies and reports found, not every interaction.
| Medicine named | Section | What the label says |
|---|---|---|
| Levodopa | drug interactions | Avoid concomitant use (e.g. sulfasalazine or sulfamethoxazole). ( 7.1 ) Interaction with Levodopa: |
| Methotrexate | drug interactions | DRUG INTERACTIONS Dihydrofolate Reductase (DHFR) Inhibitors: Avoid concomitant use (e.g., trimethoprim, methotrexate, trimetrexate, pemetrexed, pralatrexate, raltitrexed, or piritrexim). ( 7.1 ) Sepiapterin Reductase (SR) Inhibitors: |
| Pemetrexed | drug interactions | DRUG INTERACTIONS Dihydrofolate Reductase (DHFR) Inhibitors: Avoid concomitant use (e.g., trimethoprim, methotrexate, trimetrexate, pemetrexed, pralatrexate, raltitrexed, or piritrexim). ( 7.1 ) Sepiapterin Reductase (SR) Inhibitors: |
| Pralatrexate | drug interactions | DRUG INTERACTIONS Dihydrofolate Reductase (DHFR) Inhibitors: Avoid concomitant use (e.g., trimethoprim, methotrexate, trimetrexate, pemetrexed, pralatrexate, raltitrexed, or piritrexim). ( 7.1 ) Sepiapterin Reductase (SR) Inhibitors: |
| Sildenafil | drug interactions | Monitor for signs and symptoms of hypotension with concomitant use of SEPHIENCE with drugs that affect nitric oxide-mediated vasorelaxation (e.g., PDE-5 inhibitors such as sildenafil, vardenafil, or tadalafil). |
| Sulfasalazine | drug interactions | Avoid concomitant use (e.g. sulfasalazine or sulfamethoxazole). ( 7.1 ) Interaction with Levodopa: |
| Tadalafil | drug interactions | Monitor for signs and symptoms of hypotension with concomitant use of SEPHIENCE with drugs that affect nitric oxide-mediated vasorelaxation (e.g., PDE-5 inhibitors such as sildenafil, vardenafil, or tadalafil). |
| Trimethoprim | drug interactions | DRUG INTERACTIONS Dihydrofolate Reductase (DHFR) Inhibitors: Avoid concomitant use (e.g., trimethoprim, methotrexate, trimetrexate, pemetrexed, pralatrexate, raltitrexed, or piritrexim). ( 7.1 ) Sepiapterin Reductase (SR) Inhibitors: |
| Vardenafil | drug interactions | Monitor for signs and symptoms of hypotension with concomitant use of SEPHIENCE with drugs that affect nitric oxide-mediated vasorelaxation (e.g., PDE-5 inhibitors such as sildenafil, vardenafil, or tadalafil). |
Source: FDA drug label (Sephience), PTC Therapeutics Inc., effective April 24, 2026 (SPL set 32ac719c-49f0-4105-9c46-18c19583a5c2), via openFDA; the full label on DailyMed
What do FAERS reports show for sepiapterin?
The FDA Adverse Event Reporting System holds 52 reports that list sepiapterin among the medicines taken, through July 30, 2026, each a report of something that happened, not proof the medicine caused it; 0 are coded as a death and 2 as a hospital stay. Counts follow how widely a medicine is used and watched, and they are not a measure of how risky it is.
The most reported reactions
Source: FDA Adverse Event Reporting System (FAERS) via openFDA, reports through July 30, 2026
What next?
- Sepiapterin side effects
- the label's list beside FAERS reports
- Check an interaction
- what two or more labels say about each other