Source: Prism (ID: lengjing_qqfinance), Wall Street Journalist
Edit | Yang Wei
A rare type of tumor drug has recently been approved by the US Food & Drug Administration (FDA) and has been interpreted to have a 75% higher rate of treatment for multiple cancers. This has caused great concern among people, but that is not the case.
A licensed drug called Larotrectinib (commercial name Vitkravi) has been developed jointly by the precision medical company "Loxo Tumor" and the traditional pharmaceutical manufacturer "Bayer". Bayer said the drug was the first anti-tumor chemistry approved by the US Food and Drug Administration "unlike tumor types."
The US Food and Drug Administration said the newest approved drug would inhibit abnormal NTRK gene fusion with other genes – a compound that is seen as a growth-enhancing signal that supports tumor development. NTRK fusion is not common and usually occurs only in carcinogenic parts of the body, including lung cancer and thyroid cancer.
It is incorrect to understand 75% of the treatment rate
According to Bayer, the total response rate (ORR, response rate) was 75% of adults and children involved in the experiment, including 22% complete remission and 53% partial relief. The so-called remission rate refers to the proportion of people who have decreased or disappeared after the patient receives treatment, and does not refer to the proportion of the drug.
According to experimental data, a total of 55 patients participated in three trials. The US Food and Drug Administration said the experimental response was viable: from the analysis of experimental results, 73% of the relief lasted for at least six months, and 39% of the relief lasted for at least a year. Approved drugs are effective for soft tissue sarcoma, gall bladder cancer, fibrosarcoma, thyroid cancer, and lung cancer.
Although new medicines approved can treat various types of cancer, known as "broadband anticancer drugs", they are effective only for several patients with certain genetic characteristics. Accordingly, the US Food and Drug Administration also provides him with a "qualification for liver medication", according to relevant legislation, liver medications can get favorable conditions such as quick approval, tax cuts, exemption from subscription fees, research subsidies and development, etc., which will encourage pharmaceutical companies to assist and encourage the development of drugs for the treatment of rare diseases.
Specifically, only those patients with "NTRK gene fusion in tumors" are entitled to treatment with this recently-approved drug. According to Bayer, the relevant diagnosis can be diagnosed by New Generation Sequencing Technology (NGS) and Fluorescent In situ Hybridization (FISH).
In a press release, Bayer quoted Robert LaCaze, head of the Oncology Strategy Business Unit, who says: "NTRK fusion is rare, but there are many types of tumors … for those with NTRK genome fusion. It is very meaningful to provide a specialized treatment plan for patients with advanced solid tumors. "
According to Bayer, the US Department of Food and Drug Administration "approves the overall rate of response and remission duration based on a tumor drug" and approves it for "adult and boys of solid tumor patients" according to an accelerated procedure. He also noted that "subsequent approval may depend on the validation and description of the clinical benefits in the confirmatory test".
Scott Gottlieb, the US Food and Drug Commissioner, said the approval of a new drug reflects biomarker advancement in drug development and precision drug use.
High price, difficult to sell
Loxo tumors and Bayer have reached a collaboration agreement in November 2017 to develop two drugs: Larotrectinib and a new generation of TRK LOXO-195 cancer inhibitors.
As for the division of labor, Loxo tumors will conduct later clinical research and regulatory issues in the United States while Bayer is dominated by regulatory issues outside of the United States and global business activities. In the United States, both sides will jointly promote the activities of market promotion.
This is not a bargain business partnership: if co-operation is good, Bayer will pay a total of $ 1.55 billion to Loxo Cancer.
According to the agreement, Bayer will pay a $ 400 million advance on Loxo's tumor when Larotrectinib gets regulatory approvals on a particular major market and finishes its first commercial sale, the latter will receive another $ 450 million. When the LOXO-195 completes the same steps, it will receive another $ 200 million.
In terms of expenditures, two companies will share research and development costs, and in the United States two will share business expenses and equalize profits. In the US market, Bayer will pay $ 25 million when the net sales reach a certain threshold, and in the US outside the US, two companies will use a layer-by-step progressive sales approach, and Lox tumors will receive up to $ 475 million. .
Reuters quoted the financial company BTIG analyst Dane Leone, who said that Larotrectinib's annual sales would amount to $ 1.5 billion in 2026. The Financial Times quoted Citigroup's analysis, saying the annual drug sales would be in 2030 or about $ 1.9 billion.
The new approved drug is expensive and Forbes quoted Bayer as saying that for adult capsules, the price of 100 mg capsules per day for 30 days was $ 32,800. The dosage form for adults or children is $ 11,000 a month.
Earlier, some analysts believe that the two companies will face marketing difficulties. Dr. Yu, a botanic doctor at the University of Beijing, is mentioned in the popular scientific article on cancer that only 0.21% of approximately 11,000 cancer patients belong to TRK fusion carcinoma. On the other hand, related detection techniques are not currently popular and it is not difficult to find suitable patients.
Introduce the process of approving the Chinese market or accelerate
When Larotrectinib was launched in the United States, US foods and medicines selected procedures such as a Priority Review, Prosthodontic Therapy and Accelerated Approval. In fact, the agency has also widely published the drug approval process as a model to improve its effectiveness.
Earlier, the new US government promised to further simplify the oversight of the Food and Drug Administration. New Director of Food and Drug Administration Gottlieb has publicly stated that it is necessary to speed up drug approval, encourage the promotion of prescription drugs, and relax surveillance of generic drugs, as evidenced by the approval of Larotrectinib.
The rationale behind the US Food and Drug Administration's approval of medicines has long been criticized. Critics say a long-term review delayed the treatment of patients and, on the other hand, affected the commercial schedule of drug companies and increased costs. In response, the agency has tried to simplify and relax the regulation.
Medications that receive the approval priority often "significantly improve the safety, efficacy and effectiveness of treatment, diagnosis or prevention of serious conditions" compared to medicines that follow the standard inspection procedure. Approval time is shortened from 10 months to 6 months.
Acceptance of the breakthrough therapy may be used when a developing drug is used to treat a serious condition and the initial clinical outcome indicates that it has a "significant improvement" at the "clinically significant" endpoint.
In the process of accelerated approval, food and drug control, it may use the substitution endpoint or the midpoint of drug approval. In other words, when these indicators can predict that the drug is likely to have efficacy or clinical benefit, you should conditionally approve the drug on the market.
According to Bayer, he will provide patients with oral capsules and solutions on the US market, and the company filed an application for entry into the European Medicines Agency in August. As far as the arrangements for listing in China are concerned, Bayer has just said that he will continue to fulfill his commitment to introduce innovative and leading products.
Industrial people have told Tencent "Pixel" that if Bayer really intends to import related products in China, it is expected that "approval time will not be very long". The reason for this is that Chinese regulator agencies are also trying to improve drug delivery efficiency.
This is illustrated by the approval procedure for "Anticancer drug broad spectrum" Pablo monoclonal antibody. The cancer drug produced by Merck is listed in China in July 2018, and the approval process lasted less than half a year. In a press release, Merck quoted the words of his Chinese president, Luo Wanli, and praised the practice of Chinese regulators actively introducing innovative medicines and increasing the speed of approving new medicines.
Improving workflow is one of the measures. Earlier, the accounting firm Deloitte said in a report that for innovative medicines, the Chinese government's "strict admission" permission allowed the approval of clinical trials to be longer than other markets such as the United States.
In addition, local authorities are also working hard. The Shanghai Municipal Government announced on July 10 that the "100 extension width" action plan was mentioned in order to cope with the urgent need for clinically available antitumor drugs that are listed abroad and are not approved for registration in China and used in Shanghai. .
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