The National Medical Products Administration (NMPA) has initiated a pilot program to implement “Priority review and approval procedures applied to formula foods for rare diseases and special medical purposes that are urgently needed in clinical practice.” According to the People’s Daily, these procedures are expected to be incorporated into the Measures for Registration Administration of Formula Food for Special Medical Purposes. Notably, the review and approval timeframe for formulas for special medical purposes in rare diseases will be reduced to 30 working days from the previous 60, with on-site inspection and sampling inspection being given priority.
Special Medical Purpose Foods: Meeting the Needs of Those with Special Dietary Requirements
Foods for Special Medical Purpose (FSMP) are specially processed and prepared to cater to the unique nutritional needs of individuals with limited eating, digestive and absorption disorders, metabolic disorders, or specific disease states. These foods play a critical role in managing health conditions that require tailored nutritional support.
Rare Diseases and the Need for FSMP
Among the 121 rare diseases listed by the government, 32 necessitate the use of FSMP, including 18 that require timely, lifelong, and sufficient use of FSMP which cannot be substituted by existing registered enteral nutrition preparations. These diseases include:
- Amino acid metabolic diseases: complete carboxylase synthase deficiency, maple syrup urine disease, albinism, tetrahydrobiopterin deficiency, phenylketonuria, β-Ketothiolyase deficiency (β-Ketothiolase deficiency; BKD), hyperhomocysteinemia, arginase deficiency, hyperphenylalaninemia;
- Organic acid metabolism diseases: methylmalonic acidemia (MMA), isovaleric acidemia (IVA), 3-hydroxy-3-methylglutarate uria (HMG), 3-methylcrotonyl coenzyme carboxylase (MCC) deficiency;
- Fatty acid oxidative metabolism disorders: multiple acyl CoA dehydrogenase deficiency (MADD), long chain 3-hydroxyacyl CoA dehydrogenase deficiency (LCHADD);
- Carbohydrate metabolism: hereditary fructose intolerance, galactosamia;
- Severe myoclonic epilepsy in infants.-Fineline Info & Tech
