Cancer Drug Inspired by Sea Animal Gets FDA Green Light

The Spanish company PharmaMar’s stock price has shot up by 30% as its cancer drug lurbinectedin was given accelerated approval for the second-line treatment of metastatic small cell lung cancer in the US this week.

Lurbinectedin will be marketed under the brand name Zepzelca and will be made commercially available in the US in early July by PharmaMar’s partner, Jazz Pharmaceuticals. The accelerated approval was based on results from a phase II trial where lurbinectedin shrank the tumors in 35% of patients with relapsed metastatic small cell lung cancer — an aggressive form of lung cancer with a five-year survival rate of just 14%.

Drugs granted accelerated approval by the FDA typically need phase IV confirmatory trials to confirm the drug’s benefits before getting full approval. For reaching the accelerated approval stage with lurbinectedin, Jazz will pay PharmaMar €89M and will hand over an additional €134M when full approval is achieved. PharmaMar could also receive royalties of up to 30% from the drug’s sales.

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Lurbinectedin is a synthetic, modified form of a small molecule found in the sea squirt Ecteinacidia turbinata. The drug works by blocking gene expression carried out by a protein called RNA polymerase II, which many cancers depend on. While PharmaMar hasn’t carried out a direct trial comparing it with standard second-line treatments such as Topotecan and chemotherapy in this indication, the company is confident that it stands up to the competition.

“We don’t have head-to-head comparison data between Zepzelca and Topotecan in small cell lung cancer,” José María Fernández, President of PharmaMar, told me. But, an indirect comparison between Zepzelca data and the historical Topotecan data in a similar population of patients with relapsed small cell lung cancer suggests that Zepzelca offers a therapeutic advantage in terms of both efficacy and safety over Topotecan.” 

Fernández told me that the fact that the company has never directly compared Zepzelca with other drugs in small cell lung cancer was a challenge, not only in developing Zepzelca, but also in approaching regulatory agencies to request approval for this drug.

This latest approval represents a promising turn in PharmaMar’s fortunes surrounding lurbinectedin. Only two years ago, the drug failed to improve the survival of ovarian cancer patients when compared to standard second-line treatments. However, Jazz Pharmaceuticals saw promise in the drug for the treatment of small cell lung cancer, signing an €890M deal to commercialize lurbinectedin in the US late last year.

“We are very pleased and proud to be able to bring Zepzelca as a new treatment option for patients with relapsed or recurrent small cell lung cancer, at least in the USA for now. And we are making all efforts and discussing with regulatory authorities around the world in order to make Zepzelca available to all small cell lung cancer patients in need,” said Fernández.


Image from Shutterstock

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