Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

Sepiapterin - PTC Therapeutics

Next Previous
Drug Profile

Sepiapterin - PTC Therapeutics

Alternative Names: CNSA 001; PTC-923; Sephience

Latest Information Update: 24 Feb 2026

Price :
$50 *
Note:
  • Adis is an information provider. We do not sell or distribute actual drugs.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
  • A link to download a PDF version of the drug profile will be included in your email receipt.

At a glance

  • Originator Censa Pharmaceuticals
  • Developer Censa Pharmaceuticals; PTC Therapeutics
  • Class Amines; Gastrokinetics; Ketones; Pteridines; Small molecules; Urologics
  • Mechanism of Action Phenylalanine hydroxylase stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Phenylketonuria
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Phenylketonuria
  • Preclinical Lupus nephritis
  • Discontinued Diabetic gastroparesis; Dystonia

Most Recent Events

  • 19 Feb 2026 PTC Therapeutics plans approval of Sepiapterin in Brazil in 2026
  • 01 Feb 2026 Registered for Phenylketonuria (In adults, In children, In adolescents, In the elderly) in Brazil (PO)
  • 22 Dec 2025 Registered for Phenylketonuria (In the elderly, In adolescents, In children, In adults) in Japan (PO)

You need to be a logged in or subscribed to view this content

Request demo (opens in a new window)
If your organization or you do not have a subscription, try one of the following: If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top