Hoth Therapeutics Reports Orphan Designation Application to Treat Mastocytosis, To Receive Exclusive Rights and Cost Benefits on Approval

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Hoth Therapeutics, Inc. (NASDAQ: HOTH) announced that it has submitted an Orphan Drug Designation Application to the U.S. Food and Drug Administration (FDA) for HT-KIT for the treatment of mastocyctosis. HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frameshifting, resulting in apoptosis of neoplastic mast cells.

The KIT signaling pathway is implicated in multiple diseases, including all types of mastocytosis (such as aggressive systemic mastocytosis (ASM), mast cell leukemia (MCL), and systemic mastocytosis with associated hematological neoplasm (SM-AHN), acute myeloid leukemia, gastrointestinal stromal tumors, and anaphylaxis.

Drugs intended to treat orphan diseases (rare diseases that affect less than 200,000 people in the U.S.) are eligible to apply for Orphan Drug Designation (ODD), which provides multiple benefits to the sponsor during development and after approval. Hoth intends to pursue these benefits as part of the drug development for HT-KIT for treatment of mastocytosis, pending designation of the ODD application.

Benefits of Orphan Drug Designation

Under the Orphan Drug Act, drug companies can apply for ODD, and if granted, the drug will have a status that gives companies exclusive marketing and development rights along with other benefits to recover the costs of researching and developing the drug.

A tax credit of 50% of the qualified clinical drug testing costs awarded upon drug approval is also possible. Regulatory streamlining and provide special assistance to companies that develop drugs for rare patient populations.

In addition to exclusive rights and cost benefits, the FDA will provide protocol assistance, potential decreased wait-time for drug approval, discounts on registration fees, and eligibility for market exclusivity after approval.

Key benefits of ODD:

• 7 years exclusivity post-approval
• Tax credits of 50% off the clinical drug testing cost awarded upon approval
• Waiver of new drug application (NDA)/ biologics license application (BLA) application fee 

Hoth recently announced that its novel anti-cancer therapeutic exhibited highly positive results in humanized mast cell neoplasm models, representative in vitro and in vivo models for aggressive, mast cell-derived cancers such as mast cell leukemia and mast cell sarcoma.

The preceding post was written and/or published as a collaboration between Benzinga’s in-house sponsored content team and a financial partner of Benzinga. Although the piece is not and should not be construed as editorial content, the sponsored content team works to ensure that any and all information contained within is true and accurate to the best of their knowledge and research. This content is for informational purposes only and not intended to be investing advice.