The FDA has granted orphan drug status and a rare pediatric disease designation to Denali Therapeutics pipeline candidate, DNL310. The pre-clinical candidate is being evaluated for the treatment of patients with mucopolysaccharidosis II (MPS II), also known as Hunter Syndrome, which is a lysosomal storage disease caused by the IDS enzyme deficiency.
Denali plans to begin a phase I/II study on DNL310 for treating Hunter Syndrome in 2020.
In earlier pre-clinical studies, DNL310 demonstrated an increased brain uptake in a relevant disease model and robust reduction of downstream markers of disease in the central nervous system compared with the standard enzyme replacement therapy (ERT).
Orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. This designation also makes the company entitled to certain other benefits including tax credits related to clinical trial expenses and an exemption from the FDA user fee. The status will also make DNL310 eligible for seven years of marketing exclusivity in the United States.
Additionally, the rare pediatric disease designation granted to DNL310 provides Denali with an opportunity to obtain a pediatric disease Priority Review Voucher from the FDA after the candidate’s qualifying new drug application (NDA) is approved for the treatment of any rare pediatric disease. The company may use the voucher to procure a priority review for an NDA submitted later.