Abstract:
The U.S. Food and Drug Administration (FDA) approved Revolution on Wednesday Medicines’ marketing application for breakthrough pancreatic cancer drug. Large clinical trials showed the drug nearly doubled the median survival of patients with advanced pancreatic cancer. The once-daily oral drug, called Rasonque, is suitable for patients with metastatic pancreatic cancer who have received prior treatment or who are unable to receive combination chemotherapy. The drug is designed to block multiple forms of the RAS protein. RAS proteins drive tumor growth in many types of pancreatic cancer.
Specifically, patients who took Rasonque had a median survival of 13.2 months, compared with 6.7 months for those who received chemotherapy, an increase of 6.5 months.
Data show that pancreatic cancer is one of the cancers with the highest mortality rate, and the overall 5-year survival rate is among the lowest among all types of cancer, only about 13%.
今年4月，Revolution公布该药临床试验结果后，市场需求迅速升温。FDA随后迅速通过其"同情用药计划"提前向患者开放该药。该计划允许患有严重或危及生命疾病的患者，在药物获得监管机构正式批准前，于临床试验之外接受实验性治疗。
“Rasonque will change patients’ lives,” said Carla Kurkjian, an oncologist at Mercy Hospital in Oklahoma City. “This not only means that patients are expected to live longer, even to a degree that we have never seen in the past; for us doctors, it also means that we can finally have a different treatment conversation with patients than in the past few decades.”
Mark Goldsmith, CEO of Revolution Medicines, said: "This is a landmark opportunity for patients and their families, and we need to do everything we can to clear the way and make this happen as quickly as possible."
The review speed has been greatly accelerated
Last year, Rasonque became one of the first drugs to be included in the FDA's new expedited review mechanism. This process is designed to significantly compress the conventional 10 to 12 month review cycle to only 1 to 2 months, targeting treatments that address major U.S. health priorities and have huge unmet medical needs.
Market investors have been optimistic about Rasonque’s business prospects, betting that it will become an important new treatment option, pushing Revolution’s stock price up 166% this year. Since the market had generally expected the drug to be approved, the reaction to Revolution's stock price was relatively muted after the news was announced, rising only 1% during the session.
Wall Street expects Rasonque to become a blockbuster drug. Evaluate, a pharmaceutical industry business intelligence company, predicts that if the drug is proven to be safe and effective in earlier stages of the disease and other cancer types, its annual sales could exceed $20 billion.
RBC Capital Markets analysts said that the rapid approval and more than 2,000 patients currently participating in the drug's expanded use plan may push the drug's pancreatic cancer sales in the United States to approximately $28 million in the third quarter; sales in the fourth quarter may reach $148 million.
In the longer term, RBC Capital Markets predicts that the drug's annual sales may reach up to $11.5 billion.
The American Cancer Society predicts that approximately 67,350 people in the United States will be diagnosed with pancreatic cancer this year. For a long time, these patients lacked effective treatment options and were often relegated to chemotherapy. About seven out of eight patients die within five years of diagnosis, and many of them don't even survive a year.
RBC Capital Markets analysts said: "Given the excellent clinical data demonstrated by the drug and the huge unmet medical needs, the market is likely to be able to accept its higher pricing."
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