- The European Commission (EC) granted orphan designation to Timber Pharmaceuticals' ( NYSE: TMBR ) TMB-001 to treat autosomal recessive congenital ichthyosis (ARCI).
- Ichthyosis is a condition which causes persistent thick and dry skin.
- The European Medicines Agency's (EMA) Committee for Orphan Medicinal Products in September had recommended granting the drug orphan status.
- The company added that European Medicines Agency (EMA) Pediatric Committee (PDCO) also provided positive comments on the Pediatric Investigation Plan (PIP) to treat ARCI and X-linked recessive ichthyosis (XLRI).
- This has set the basis for a final discussion in mid-November, which is requirement for filing a marketing authorization application (MAA) with the EMA, the company noted.
- Timber said TMB-001 is a topical isotretinoin, formulated using the its patented IPEG delivery system.
- TMB-001 is being evaluated in a phase 3 trial called ASCEND.
- The orphan drug status in the EU is aimed at therapies for treating or preventing diseases which affect fewer than five in 10,000 people in the EU. The designation provides incentives, including 10 years' of market exclusivity for the drug, if approved.
For further details see:
Timber's skin disorder therapy TMB-001 gets orphan drug status in EU