On October 21, 2025, Hoth Therapeutics announced that the U.S. Food and Drug Administration granted orphan drug designation to its precision antisense oligonucleotide, HT‑KIT, for rare c‑KIT‑driven cancers such as systemic mastocytosis and gastrointestinal stromal tumor (GIST). The designation was announced today in a press release issued by the company.
The company reported that HT‑KIT achieved more than 80 % suppression of KIT mRNA and produced a significant reduction in tumor volume in both systemic mastocytosis and GIST models by Day 8 of treatment. In addition, the company completed GLP‑validated bioanalytical methods that will support IND‑enabling studies, and it highlighted a clean preclinical tolerability profile across all models tested.
Hoth also noted that its Japan Patent No. 7677628, granted in 2025, extends platform protection through 2039 and that the IND‑enabling package is progressing toward first‑in‑human evaluation. The company’s CEO, Robb Knie, emphasized that the orphan designation and the robust preclinical data position HT‑KIT for accelerated regulatory review and potential market exclusivity under the orphan drug act.
The orphan drug status is a material benefit for Hoth, providing up to seven years of market exclusivity, eligibility for tax credits, and priority review, all of which can reduce time to market and improve the commercial viability of HT‑KIT. This regulatory milestone also strengthens the company’s pipeline and may enhance investor confidence by demonstrating tangible progress toward a first‑in‑class therapy for a high‑need patient population.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.