Experimental Treatment for Hunter Syndrome Earns Rare Pediatric Disease Designation and Orphan Drug Designation

According to a story from globenewswire.com, the biopharmaceutical company Denali Therapeutics, Inc. has recently announced that its experimental product candidate DNL310, which is currently being developed as a treatment for Hunter syndrome, has earned both Rare Pediatric Disease designation and Orphan Drug designation from the US Food and Drug Administration (FDA). Denali is focused on the development of new therapies for neurodegenerative diseases.

About Hunter Syndrome (MPS II)

Hunter syndrome, which is also known as mucopolysaccharidosis II (MPS II), is a rare lysosomal storage disease. This X-linked disorder is caused by mutations of the IPS gene and affects males almost exclusively. This mutation causes a deficiency of a certain enzyme which allows other substances to accumulate in the body and cause symptoms. These symptoms include ear infections, abdominal hernia, enlarged tongue, distinct facial features, developmental declines, joint stiffness, enlarged spleen and liver, heart problems, skin lesions, behavioral abnormalities, retinal degeneration, and hearing loss. Treatment for Hunter syndrome includes enzyme replacement therapy and bone marrow or stem cell transplantation; these treatments cannot address neurological symptoms. Research into gene therapy for this disease is also ongoing. There are around 2,000 people worldwide that are known to have the disorder. To learn more about Hunter syndrome, click here.

About Orphan Drug Designation

Orphan Drug designation is typically reserved for drugs that are being developed to treat diseases that are considered rare, which is defined as any disease with less than 200,000 patients in the US. To qualify, a therapy must demonstrate significant advantages in either safety or effectiveness compared to current treatments or satisfy a currently unmet medical need. This designation confers several benefits for the recipient company, such as tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the drug gains approval.

About Rare Pediatric Disease Designation

Rare Pediatric Disease designation may be granted for therapies that are being developed for a rare disease (less than 200,000 patients) that also disproportionately affects people that are under the age of 18. If a drug with this designation is approved, the company is awarded a Priority Review voucher that it can use with a future product.

Denali also announced that two more of its candidates have advanced to the pre-clinical testing stage.


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