phenylketonuria

CHMP meeting highlights – April 2025

28 April 2025 | By Catherine Eckford (European Pharmaceutical Review)

The Committee for Medicinal Products for Human Use (CHMP) recommended approval of a variety of new medicines, including several treatments for hereditary and rare conditions.

news

FDA grants Orphan Drug Designation to phenylketonuria treatment

7 April 2020 | By Victoria Rees (European Pharmaceutical Review)

A new treatment called APR-OD031 has been given Orphan Drug Designation (ODD) by the FDA, for the treatment of phenylketonuria.

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phenylketonuria

CHMP meeting highlights – April 2025

FDA grants Orphan Drug Designation to phenylketonuria treatment