The FDA on Monday approved a new drug for patients with advanced small cell lung cancer, permitting the treatment to be marketed based on less evidence than it traditionally requires in order to speed its path to market.
Madrid-based PharmaMar, which developed lurbinectedin (Zepzelca), licensed its US commercialization rights to Jazz Pharmaceuticals (NASDAQ: JAZZ) last December.
Now Jazz, which is based in Dublin, Ireland, but has significant US operations in Silicon Valley and Philadelphia, is preparing to make the drug a treatment option for adults whose small cell lung cancer (SCLC) has spread to other parts of the body and has not or is no longer responding to treatment with platinum-based chemotherapy. Few treatment options are available to such patients.
Lung cancer is the top cause of cancer death in the US. Small cell lung cancer, which is closely associated with tobacco smoking, makes up about 10 percent to 15 percent of all lung cancers, according to the American Cancer Society. While less common than the other main type of the disease, non-small cell lung cancer, it’s much more aggressive—and, the majority of the time, once a patient is diagnosed the cancer has already spread. About 30,000 new SCLC cancer cases are diagnosed yearly in the US.
The FDA’s approval of lurbinectedin, like a number of others granted recently, came months early. The agency’s deadline for a decision was Aug. 16.
The agency’s review was based on data from an open-label Phase 2 trial that enrolled 105 patients. Investigators reported that 35 percent of patients demonstrated an overall response rate, meaning their tumor size shrank by a predefined minimum amount for a predetermined amount of time, and that the response lasted for a median duration of 5.3 months. An independent review committee found that 30 percent of patients exhibited a response and that its median duration was 5.1 months.
Data on tumor response, which doesn’t always correlate with survival, are among the indirect signs of clinical benefit that the FDA considers when reviewing potential drugs for accelerated approval. For Jazz to maintain its permission to market the drug, the FDA is requiring the company to conduct additional testing to verify that the drug confers clinical benefit.
Lurbinectedin, which in the trial was delivered via an hour-long infusion every three weeks, is what’s known as an alkylating drug. The compound attacks DNA in a way that’s intended to disrupt the growth and division process and eventually cause cell death.
Common side effects included low white and red blood cell counts, liver problems, tiredness, increased blood sugar, nausea, decreased appetite, muscle and joint pain, low level of albumin in the blood, constipation, trouble breathing, low levels of sodium and magnesium in the blood, vomiting, cough, and diarrhea. Delivery of the drug was discontinued in 1.9 percent of patients due to side effects and delayed in 30.5 percent. Side effects prompted a reduction in the dose for 25 percent of study participants.
Jazz anticipates the drug will become available in the US in early July.
Last year net product sales from Jazz’s cancer and hematology drugs brought in $472 million, compared to $1.6 billion for the sleep and neuroscience-related treatments for which it’s best known. The cancer drugs in the Jazz portfolio are leukemia treatments. The most recent addition was Vyxeos, a reformulation of the chemotherapy combination of cytarabine and daunorubicin, which Jazz added in 2016 when it acquired Celator Pharmaceuticals for $1.5 billion.
This year the company anticipates revenue from sales of its cancer and hematology drugs could reach up to $510 million, assuming a third-quarter launch of lurbinectedin, according to its latest guidance. Recently the company lowered guidance for those segments from from up to $580 million, citing uncertainties related to the COVID-19 outbreak.
Jazz paid PharmaMar $200 million up front for US rights to the drug. It also agreed to pay up to $250 million more in milestones tied the drug’s approval and up to an additional $550 million in connection with commercialization, plus royalties.
Lurbinectedin is also under evaluation in combination with the chemotherapy doxorubicin in a PharmaMar-funded Phase 3 randomized trial. PharmaMar could also get additional fees if the drug is approved for other indications. Jazz R&D chief Rob Iannone recently said the company is considering advancing the drug as a first choice for treating SCLC patients.
Company shares closed Monday at $110.50, up 5 percent from market close Friday.