NEW YORK – Hoth Therapeutics, Inc. (NASDAQ: HOTH) a patient-focused clinical-stage biopharmaceutical company, announced it submitted an Orphan Drug Designation Request to the US Food and Drug Administration (FDA) for HT-KIT for the treatment of mastocytosis.
HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frameshifting, resulting in the 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 US) 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 Request.
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.
About Hoth Therapeutics, Inc.
Hoth Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing new generation therapies for unmet medical needs. Hoth’s pipeline development is focused to improve the quality of life for patients suffering from indications including atopic dermatitis, skin toxicities associated with cancer therapy, chronic wounds, psoriasis, asthma, acne, and pneumonia. Hoth has also entered into two different agreements to further the development of two therapeutic prospects to prevent or treat COVID-19. To learn more, please visit www.hoththerapeutics.com.
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