Source: Prisma (ID: lengjing_qqfinance), Wall Street Journalist
Edit | Yang Wei
A treatment for a rare type of tumor has recently been approved by the US Food & Drug Administration (FDA) and has been interpreted to have a 75% cure rate for some types of cancer. This has caused great concern among people, but this is not the case.
The approved drug, called Larotrectinib (trade name Vitkravi), was jointly developed by the precision medical company "Loxo Tumor" and the traditional pharmaceutical manufacturer "Bayer". Bayer said the drug was the first anti-tumor chemical approved by the US Food and Drug Administration because "there was no difference between the types of tumors."
The US Food and Drug Administration has said that the last approved drug will inhibit abnormal fusion of the NTRK gene with other genes – a fusion that has been seen as leading to growth signals that support tumor development. NTRK fusion is not common, and usually only occurs in cancerous body parts, including lung cancer and thyroid cancer.
The cure rate is 75% misunderstood
According to Bayer, the overall response rate (ORR, translation response rate) was 75% among adults and children involved in the trial, including 22% complete remission and 53% partial assistance. The rate of remission refers to the proportion of people who have dropped or disappeared after the patient has received treatment, and does not refer to healing proportions.
According to experimental data, 55 patients participated in three trials. The US Food and Drug Administration says that the experimental response is ongoing: in the analysis of experimental results, 73% of assistance lasts at least six months, and 39% of assistance lasts at least one year. Effective drugs for soft tissue sarcoma, salivary gland cancer, infant fibrosarcoma, thyroid cancer, and lung cancer.
Although new approved drugs can treat a variety of cancers, known as "broad-spectrum anticancer drugs," they are only effective for some patients with certain characteristics at the genetic level. Therefore, the US Food and Drug Administration also gives it "orphaned drug qualifications" – according to relevant laws, orphan drugs can obtain comfortable conditions such as fast approvals, tax deductions, exemptions from reporting costs, research and development subsidies , etc., which will provide incentives for pharmaceutical companies to help and encourage the development of medicines to treat rare diseases.
In particular, only patients with "NTRK gene fusion in tumors" were eligible for treatment with this newly approved drug. According to Bayer, relevant diagnoses can be diagnosed using next generation sequencing technology (NGS) and fluorescence in situ hybridization (FISH).
In a press release, Bayer quoted Robert LaCaze, head of the oncology strategy business unit, as saying, "NTRK fusion is rare, but exists in various types of tumors … for those with NTRK gene fusion. This is very meaningful to provide patients with specific care plans with advanced solid tumors. "
According to Bayer, the US Food and Drug Administration "approved the overall response rate and duration of remission based on drugs for the treatment of tumors" and agreed to "solid adult and child tumor patients" in accordance with accelerated procedures. It also states that "further agreement may depend on the validation and description of clinical benefits in the confirmation trial".
US Food and Drug Administration Commissioner Scott Gottlieb said the approval of the new drug reflects the progress of biomarkers in guiding drug development and more appropriate drug use.
High prices, difficult to sell
Loxo Tumors and Bayer reached an exclusive cooperation agreement in November 2017 to develop two drugs: Larotrectinib and the next generation TRK cancer inhibitor LOXO-195.
In terms of division of labor, Loxo's tumors will lead clinical research and subsequent regulatory problems in the United States, while Bayer dominates regulatory issues outside the United States and global business activities. In the United States, both parties will jointly promote market promotion activities.
This is not a cheap business partnership: if cooperation goes well, Bayer will pay a total of $ 1.55 billion to Loxo Cancer.
Under the agreement, Bayer will pay a $ 400 million advance for Loxo tumors, when Larotinib gets regulatory approval in certain major markets and completes the first commercial sale, the latter will receive another $ 450 million. When LOXO-195 completes the same steps, it will receive another $ 200 million.
In terms of expenditure, both companies will share the costs of R & D, in the United States, both will share business expenses and equalize profits. On the US market, Bayer will pay $ 25 million when net sales reach a certain threshold, in the US outside the US, the two companies will use a progressive approach to layer for sale, and Loxo tumors will receive up to $ 475 million. .
Reuters quoted financial company BTIG analyst Dane Leone as saying that Larotrectinib's annual sales would peak in 2026, at $ 1.5 billion. The Financial Times cited Citigroup's analysis as saying that annual drug sales would peak at 2030, or around $ 1.9 billion.
The newly approved drug is expensive, and Forbes quoted Bayer as saying that for adult capsules, the price of a 100 mg capsule per day for 30 days is $ 32,800. The dosage form for adults or children is $ 11,000 per month.
Previously, some analysts believed that the two companies would face marketing difficulties. On the one hand, the proportion of patients with this disease is limited to cancer patients, Dr. Yu, a botanical doctor at Peking University, mentioned in a popular science article about cancer which is only 0.21% of about 11,000 cancer patients included in cancer fusion TRK. On the other hand, related detection techniques are currently unpopular, and it is not difficult to screen appropriate patients.
Introducing the Chinese market approval process or accelerating
When Larotrectib was launched in the United States, the US Food and Drug Administration chose procedures such as Priority Reviews, Breakthrough Therapy, and Accelerated Approval. In fact, agencies also publicly publish the drug approval process as a model to improve efficiency.
Previously, the new US government promised to further simplify the supervision of the Food and Drug Administration. The new Director of the Food and Drug Administration Gottlieb openly stated that the need to speed up drug approvals, support the promotion of over-the-counter drugs, and relax the supervision of generic drugs. This is proof that Larotrectinib is approved.
Wise procedures from the US Food and Drug Administration on drug approval have been criticized for a long time. Critics say that long review times delay patient treatment and on the other hand affect the commercial arrangements of drug manufacturers and increase costs. In response, agencies try to simplify and loosen regulations.
Drugs that receive priority approval often "increase significantly in the safety, efficacy, and effectiveness of treatment, diagnosis, or prevention of serious conditions" compared to drugs that follow the Standard Review process. Approval time is shortened from 10 months to 6 months.
Feasibility of breakthrough therapy may apply when the drug developed will be used to treat serious conditions and early clinical results indicate that it has "substantial improvement" at the "clinically meaningful" endpoint.
In an accelerated approval process, food and drug supervision can use substitution endpoints or intermediate points to approve the drug. In other words, when these indicators can predict that the drug is likely to have clinical benefits or benefits, conditionally approve the drug to the market.
According to Bayer, it will provide patients with capsules and oral solutions on the US market, in addition, the company has submitted a list application to the European Medicines Agency in August. As for arrangements to register in China, Bayer only said that it would continue to fulfill its commitment to introduce more innovative and leading products.
Industry people told Tencent "Pixel" that if Bayer really intends to introduce related products in China, it is hoped "the approval period will not be long." The reason is that Chinese drug regulatory agencies are also trying to improve the efficiency of drug approval.
This is illustrated by the approval process for the "Extensive Anticancer Spectrum" of monoclonal antibody Pabolim. Anti-cancer drugs produced by Merck are registered in China in July 2018, and the approval process takes less than half a year. In a press release, Merck quoted the words of its Chinese president, Luo Wanli, and highly praised the practice of Chinese regulators who actively introduced innovative drugs and increased the speed of approval of new medicines.
Improving workflow is one measure. Earlier, accounting firm Deloitte said in a report that for innovative drugs, the Chinese government's "strict entry" attitude allows approval of clinical trial applications for longer than other markets such as the US.
In addition, the regional government also works hard. The Shanghai City Government announced on July 10 that the action plan "100 expansion plan" mentions "to fight for the urgent need for clinically available anti-tumor drugs that have been registered overseas and have not been approved for registration in China, and used in Shanghai . " .
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Original title "Decrypt" broad-spectrum anticancer drug: cure rate 75% misread, applies to cheaper and harder to sell | Prism
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