FDA Grants Orphan Drug Designation to LP-284 for Soft Tissue Sarcoma

The U.S. Food and Drug Administration (FDA) granted orphan drug designation to LP-284, an investigational treatment developed by Lantern Pharma, for patients with soft tissue sarcomas, according to a news release from the company.

The designation applies in the U.S. and is intended to support the development of therapies for rare diseases with limited treatment options. The move expands LP-284’s development beyond blood cancers into solid tumors, reflecting an effort to address unmet needs among patients with soft tissue sarcomas, most of whom are adults.

"With over 79% of cases occurring in adults, this designation addresses a distinct patient population with significant unmet need,” said Panna Sharma, CEO of Lantern Pharma.

What is LP-284? How is it Being Studied in Patients with Cancer?

LP-284 is a small molecule designed to target cancers with defects in DNA repair mechanisms. It works through a synthetic lethal approach that focuses on transcription-coupled nucleotide excision repair, a pathway involved in fixing DNA damage. LP-284 belongs to the acylfulvene class of compounds and has shown activity in preclinical studies and early clinical development across multiple cancer types. According to the company, this activity has been observed regardless of TP53 mutation status or surface antigen expression.

The press release highlights clinical findings from an ongoing phase 1 trial evaluating LP-284 in B-cell non-Hodgkin lymphomas, including mantle cell lymphoma and high-grade B-cell lymphoma with MYC and BCL2 rearrangements. In July 2025, Lantern reported that a heavily pretreated 41-year-old patient with aggressive diffuse large B-cell lymphoma achieved a complete metabolic response after receiving LP-284. The patient had previously not responded to three prior treatment regimens, including standard chemo-immunotherapy, CAR-T cell therapy and CD3xCD20 bispecific antibody therapy. After enrolling in April 2025, the patient completed two 28-day cycles of LP-284 and showed non-avid lesions on imaging.

Although this clinical activity was observed in a blood cancer, Lantern stated that the same DNA repair vulnerabilities targeted by LP-284 are commonly seen in adult soft tissue sarcomas, supporting the rationale for studying the drug in this solid tumor population.

How Common are Soft Tissue Sarcomas? What Unmet Needs Exist for Patients?

Soft tissue sarcomas are a group of rare cancers that form in tissues such as muscle, fat, blood vessels, nerves, tendons and joint linings. According to data cited in the press release, approximately 13,520 new cases of soft tissue sarcoma are expected to be diagnosed in the United States in 2025. More than 79% of cases occur in patients 40 years of age or older.

Globally, soft tissue sarcomas account for about 96,200 new cases diagnosed worldwide in 2021. Across the seven major pharmaceutical markets — the United States, France, Germany, Italy, Spain, the United Kingdom and Japan — the therapeutic market for soft tissue sarcoma reached an estimated value of $2.4 billion in 2025 and is projected to grow to approximately $4.7 billion by 2035.

Adult soft tissue sarcomas differ biologically from pediatric sarcomas. While pediatric cases are often driven by specific gene fusions, adult sarcomas more commonly show complex genomic alterations, chromosomal instability and deficiencies in DNA repair pathways. The press release notes that treatment options for advanced or metastatic soft tissue sarcomas remain limited, with five-year survival rates for distant disease reported at approximately 16-17%, highlighting a significant unmet medical need for patients with cancer in this setting.

What Does Orphan Drug Designation Mean for Patients and Future Development?

The FDA’s orphan drug designation program is intended for therapies that treat rare diseases affecting fewer than 200,000 people in the United States. For drug developers, the designation provides several incentives, including seven years of market exclusivity if the drug receives regulatory approval, tax credits for qualified clinical trials, exemption from certain FDA user fees and assistance with clinical trial design.

The press release did not report specific safety outcomes for LP-284 in patients with soft tissue sarcomas, as clinical testing in this population has not yet been detailed. LP-284 is currently being evaluated in a phase 1 clinical trial registered as NCT06132503 in patients with B-cell non-Hodgkin lymphomas. Safety data from that trial were not described in detail in the announcement.

Lantern Pharma emphasized that orphan drug designation does not mean the drug is approved or proven safe and effective. Instead, it is a regulatory step intended to encourage continued research and development for patients with rare cancers, including those with soft tissue sarcomas, who currently have limited treatment options.

Reference

1. “Lantern Pharma's LP-284 Receives FDA Orphan Drug Designation for Soft Tissue Sarcomas.” News Release. Lantern Pharma. Jan 20, 2026.

Editor's note: This article is for informational purposes only and is not a substitute for professional medical advice, as your own experience will be unique. Use this article to guide discussions with your oncologist. Content was generated with AI, reviewed by a human editor, but not independently verified by a medical professional.

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