The Saudi Food and Drug Authority (SFDA) has granted orphan drug designation to Gritgen Therapeutics Co., Ltd. for its “GS1168 Injection” intended for the treatment of Phenylketonuria (PKU). This designation reflects the SFDA’s continued commitment to supporting pharmaceutical innovation and improving patient access to advanced therapies for rare diseases.

Understanding Phenylketonuria (PKU)

PKU is a rare inherited metabolic disorder caused by a deficiency or complete absence of the phenylalanine hydroxylase (PAH) enzyme activity, resulting from mutations in the gene responsible for producing this enzyme. This deficiency prevents the body from effectively breaking down phenylalanine—an amino acid found in proteins—leading to its accumulation in the blood and central nervous system.

Delayed Diagnosis May Lead to Serious Health Complications

Delays in diagnosis and therapeutic intervention can lead to serious health complications, including intellectual disability, seizures, and behavioral disorders. Other manifestations may include acquired microcephaly, hypopigmentation of the skin and hair, and a distinctive odor in the breath, skin, or urine caused by elevated phenylalanine levels in the body.

Registration is Subjected to Full Scientific Evaluation

The SFDA clarified that designation under the Orphan Drug Program allows “GS1168 Injection” to proceed within the designated regulatory pathway under specific conditions and procedures. It does not constitute final marketing authorization. The final registration decision will be announced at a later stage, following the completion of a comprehensive technical evaluation in accordance with applicable regulations.

SFDA Orphan Drug Program Accelerates Access to Critical Therapies

This approval highlights the SFDA's commitment to enhancing access to treatments for rare and chronic diseases through the Orphan Drug Program. This initiative aims to accelerate the availability of breakthrough therapies and address unmet medical needs, thereby improving healthcare quality in alignment with the Health Sector Transformation Program under Saudi Vision 2030.

Orphan drug designation applies to therapies intended for the treatment of rare diseases or conditions that affect a small segment of the population. Specifically, in the Kingdom of Saudi Arabia, this threshold is defined as conditions affecting fewer than 5 out of 10,000 individuals.

For further information about the Guidance for Orphan Drug Designation, please visit: (https://www.sfda.gov.sa/en/regulations/88482) and for inquiries, please contact: (Designation.Drug@sfda.gov.sa)