European Medicines Agency (EMA) Announces Summary of New Drug Authorizations for 2020 (II)

In 2020, as the new crown ravaged the world and humanity continued to climb the mountain in the face of adversity, the European Medicines Agency (EMA) summarized the year-round recommendations for new drug authorizations, with positive views on 97 of them, 2 negative opinions, and 16 withdrawal applications, including 39 new active substances.
01 Conditional Marketing Authorization In 2020, the EMA has approved a total of 13 conditional sales authorizations for drugs designed to enable EU patients to obtain new drugs at an early time to meet their medical needs, and conditional authorization to allow advance drug approval in cases of incomplete clinical data and replenishment after the collection of complete data in the real world.
Cancer Ayvakyt: Treatment of gastrointestinal substrate tumors (gastric and bowel cancers) that cannot be surgically removed and has spread to other parts of the body can be used when the D842V mutation, i.e. plateboard-derived growth factor α is essentially altered in cancer cells.
EMA requires the company to then provide safety and efficacy results and conduct real-world research.
Blenrep: For the treatment of recurring and resuscable multiple myeloma, the EMA requested a controlled study of Blenrep's combined low-dose dexamison with Pomadamine (Pomadamine is the best treatment for patients with multiple myeloma) to verify its efficacy and safety.
Enhertu: For the treatment of HER2-positive metastasis breast cancer, the company will provide the results of an interim trial that compares Enhertu to a traditional treatment, Retsevmo: For the treatment of cancers that cause gene fusion due to rearm (RET) during trans-infection.
EMA requires companies to provide effective and safe results in the follow-up process and to compare trials with drugs that have been authorized to treat related cancers.
Rozlytrek: Used to treat patients with solid tumors with neurotrophic tyrosine-intrinsic kinase gene fusion, or patients with advanced non-small cell lung cancer who are ROS1-positive.
company plans to submit long-term follow-up data for major studies that include the long-term effectiveness and safety of specific subgroups (older, female, and severely ill).
coVID-19, Veklury and Comirnaty have been approved for treatment of COVID-19, and the safety and versality of the product will be disclosed later.
circulatory system, Adakveo: used to prevent relapse of vascular occysts in patients with sickle cell disease (the blood vessels are blocked by abnormal red blood cells, restricting blood flow to the organs, resulting in insufficient blood supply).
the company is currently studying patient use data and will then provide the final results of the study.
anti-infection Hepcludex: Used to treat chronic hepatitis virus infection, the company said it would provide research later.
: For the treatment of drug-resistant tuberculosis in adults, the company is studying the relationship between dosage and safety.
Immunology Idefirix: For the treatment of patients who are waiting for an organ transplant but are highly sensitive to donor tissue, the company is studying the long-term survival rate and organ function of patients undergoing kidney transplants after Idefirix treatment.
, they also provide ideafirix's effectiveness and security.
Zolgensma: Used to treat spinal muscular dystrophy (SMA), a severe neurological disease that causes muscle atrophy and weakness.
the company will provide additional data on benefits and risks, including two studies of SMAI patients diagnosed with less than six months and those diagnosed with less than six weeks.
patients have not yet shown symptoms, but can be diagnosed with SMA based on genetic testing.
02 Special Cases Approval This year, the EMA approved five drugs that allow patients to obtain drugs that are not available under standard authorization, but this approval method does not provide access to complete drug data, and there may be potential risks such as the inability of certain rare diseases to be effectively evaluated, the safety and ability of collecting drugs to violate ethics, or gaps in the scientific field, so drugs are required to comply with special regulations.
Hematology Elzonris: For the treatment of plasma cellular fibroids (BPDCN) Lumoxiti: Treatment of refractic and recurring hair cell leukemia, a type of white blood cell cancer associated with B lymphocytes, both drug developers will meet their obligations to provide the effectiveness, safety demonstration, and continue to track real-world disease data.
Anti-infection ObiltoximabSFL: Treatment of inhaled anthrax caused by Bacillus anthrax, the company will verify the drug's absorption, effectiveness, metabolism in subsequent laboratory studies, and submit data on the effectiveness and safety of the drug's prevention during the incubation period of anthrax.
vaccines Mvabea and Zabdeno: are two components of the vaccine, which protects children and adults over the age of one from the Ebola virus, and the company regularly collects data on vaccine treatment options for target populations each year.
03 Priority Medicines (PRIME) EMA provides strong support for these drugs, speeding up the process of approval and helping patients benefit from them.
year, eight PRIME-specific drugs have been approved (Blenrep, Rozlytrek, Tecartus, Givlaari, Hepcludex, Zolgensma, Idefirix and Oxlumo), and 20 drugs in development have been included in the program.
04 rare disease drug EU has its own framework for rare disease drugs, which can provide incentives for developers to develop more drugs for patients.
ema's Rare Disease Drug Industry Committee (COMP) reviews the drug's approval based on the information available to determine whether it is allowed to obtain rare disease drug status, and the drug under review will gain a decade of market dominance.
22 of the 97 drugs recommended for sale in 2020 have been identified as rare disease drugs.
these include Elzonris in the field of blood circulation (treatment of plasma cellular fibroids), Hepcludex in the field of anti-infection (treatment of chronic hepatitis virus infection), Libmeldy in the neurological field (treatment of iso-infected cerebral white blood malnutrition), Oxlumo in the field of urology (treatment of rare genetic diseases of primary high herbal uremia type 1).
05 The new use of "old drugs" in 2020, EMA expanded the use of these drugs already licensed in the European Union, but also to provide patients with new treatment options, expanding the use of adaptive drugs include: endocrinology Orfadin: to treat adult patients with black Urology Dermatology Olumiant: Velphoro for the treatment of mild to severe endemic dermatitis in recognized patients with urological nephrology: Used to control serum phosphorus levels in children over 2 years of age with chronic kidney disease (CDK) Stage 4-5 or CDK dialysis.
06 is whether it is conditional marketing authorization, or special circumstances, priority drugs, rare disease drugs and other special approval process, is for patients to be able to "use drugs for the disease" as soon as possible.
security and high prices, the system is still a positive guide for us.
Drug pre-market clinical research has limitations, usually fewer cases, short research time, case age range is narrow, drug conditions are ideal, lack of sufficient reliable clinical data support, so China actively learn from the experience of developed countries, "national basic drug catalog," "over-the-counter drug catalog," "elimination of varieties catalog" and "new drug trial production period" and other policies have been introduced, for the national pharmaceutical reform pilot provides a strong support, some of the realization of the clinical precision application of drugs.
in the field of rare diseases, China is also actively learning from the management experience of developed countries.
May 2018, the five ministries jointly released the First List of Rare Diseases, covering 121 rare diseases;
more and more rare disease drugs are also included in health insurance, we are with a firm determination to defend the hope of patients with rare diseases, the future can be!