PharmaMar’s lurbinectedin granted orphan drug designation by FDA for small cell lung cancer treatment

Spanish pharmaceutical company PharmaMar has announced that its cancer drug, lurbinectedin, has received orphan drug designation from the US Food and Drug Administration (FDA) for the treatment of small cell lung cancer (SCLC). This designation marks a critical milestone in the development of lurbinectedin, highlighting its potential in treating this aggressive form of cancer.

Lurbinectedin works by inhibiting the RNA polymerase II enzyme, crucial for the transcription process that is often over-activated in tumors with transcription addiction. This unique mechanism of action sets lurbinectedin apart as it targets a fundamental process in cancer cell proliferation, offering a new approach to treating SCLC, which is known for its poor prognosis and limited treatment options due to rapid metastasis at the time of diagnosis.

The FDA grants orphan drug designation to facilitate the development of treatments for rare diseases or conditions affecting fewer than 200,000 people in the US. This status provides various development benefits, including tax credits, user fee waivers, and eligibility for seven years of market exclusivity post-approval.

Luis Mora, Managing Director of the Oncology Business Unit at PharmaMar, expressed his enthusiasm about the designation: “We are delighted to receive this orphan drug designation as it underscores the great need for innovative, effective treatments for this cancer, and recognizes the potential benefits that lurbinectedin may provide for patients with small cell lung cancer.”

PharmaMar also highlighted recent progress in the clinical development of lurbinectedin. The company successfully completed patient recruitment for the pivotal Phase 3 trial named ATLANTIS, evaluating lurbinectedin in combination with doxorubicin against other chemotherapy options. This trial has enrolled 600 patients and aims to establish a new treatment regimen for relapsed small-cell lung cancer.

With this regulatory milestone, PharmaMar continues to advance its commitment to addressing unmet medical needs in oncology. The orphan drug designation not only enhances the development process for lurbinectedin but also aligns with PharmaMar’s strategy to bring innovative cancer therapies to the market.

As the ATLANTIS trial moves forward, the oncology community watches closely, hopeful that lurbinectedin will provide a much-needed new option for treating small cell lung cancer, potentially improving outcomes for patients facing this challenging diagnosis.