"Orphan medicine" is also known as rare medicine, refers to the use of drugs for the prevention, treatment, diagnosis of rare diseases.
, rare diseases are those that affect fewer than 200,000 people.
In 1983, the United States enacted the Orphan Drugs Act (ODA), followed by the introduction of special treatment for orphan drug reviews, coupled with a 50% tax credit for clinical research and testing costs, a reduction in prescription drug user fees, and a seven-year market exclusive period after drug approval, which led to increasing social awareness of orphan drugs and growing enthusiasm between pharmaceutical companies for the development of orphan drugs.
the number of orphan drug qualifications granted by the FDA and the number of approved orphan drug adaptations are increasing year by year, according to the U.S. Drug Information Administration, which has so far granted 5,712 orphan drug eligibility and approved 930 orphan drug adaptations.
only in 2020 (as of December 17), the FDA has granted 460 orphan drug eligibility and approved 79 orphan drug adaptations.
2020 FDA approved the classification of orphan drugs (Source: Pharmaceutical Intelligence U.S. Listed Drug Database) Domestic enterprises for orphan drug research and development started late, but in recent years, a number of innovative pharmaceutical companies have joined the development of rare disease drugs.
2020, China's "orphan medicine" research and development has made a breakthrough.
so far, there are 24 new Chinese drugs that will qualify for orphan drugs in the United States by 2020.
these products, mainly anti-tumor drugs, but also anti-infection and other rare disease drugs.
, AXA Pharmaceuticals 4 products won 8 orphan drug certification, a number of PD-1/L1 inhibitor adaptation is extremely fierce.
: data for the author manually organized, there may be some omissions and errors.
PD-1/L1 inhibitor controversy intensified according to statistics, thought Di, and platinum medicine, cornerstone pharmaceutical industry, Junshi biological, Xinda biology, Baiji Shenzhou and other enterprises have PD-1/L1 inhibitors to obtain orphan drugs Qualifications, including nasopharyngeal cancer, T-cell lymphoma and other adaptations, in addition, Corning Jerry PD-L1/CTLA-4 double anti-KN046 treatment of thymus epithelial tumors also qualified by the FDA orphan drug.
1. CorningErry/Thinking DiKN035 is a fusion protein of PD-L1 mono-domain antibody Fc, developed by Corning Jerry and ThinkDy, and Synth Pharmaceuticals has exclusive rights to market and promote all of its tumor adaptations in China.
January, the FDA granted KN035 an orphan drug for advanced bile gallbladder cancer.
The product is currently conducting simultaneous clinical trials in China, the United States and Japan for a number of tumor adaptations, including Phase 2 clinical trials for colorectal cancer and other advanced solid tumors in China and Phase 3 clinical trials for advanced bilibromatic cancer in China.
according to the prospection, the product is expected to be able to submit a new drug listing application in the second half of 2020.
KN046 is a recombinant humanized PD-L1/CTLA-4 dual-specific antibody used to treat thymus epithal tumors.
September, the FDA granted the drug orphan drug eligibility, the second orphan drug eligibility for Corninger Pharmaceuticals.
2. and platinum pharmaceutical HBM9167 (also known as KL-A167) is a recombinant humanized IgG1 monoclonal antibody for programmed cell death ligand 1 (PD-L1) developed by Columbine Pharmaceuticals Holdings, and Platinum owns the global development and commercialization rights for the drug candidate outside Greater China.
, the second phase of HBM9167 was approved by the U.S. FDA and qualified as a drug for the treatment of nasopharyngeal cancer.
, the product is currently in China to treat nasopharyngeal cancer, recurrence or recurring classic Hodgkin's lymphoma, recurrence of recurring malignant lymphoma and other tumor adaptations of Phase 1 and Phase 2 clinical trials.
3. Keystone Pharmaceuticals CS1003 is a humanized recombinant IgG4 monoclonal antibody targeting PD-1 and is being developed for immunotherapy for a variety of tumors, as one of the products of Keystone Pharmaceuticals' tumor immune skeleton.
, CS1003 was awarded by the FDA to treat hepatocellular carcinoma.
, phase 1 clinical trials of CS1003 are currently being conducted simultaneously in Australia, New Zealand and China.
CS1001 (Shugli monoanti) was developed by Keystone Pharmaceuticals in the study of anti-PD-L1 monoclonal antibodies.
, the FDA granted the PD-L1 antibody Shugli monoanti (CS1001) orphan drug eligibility for the treatment of T-cell lymphoma.
three is the second orphan drug to be awarded by the FDA following the FDA's award in July of its anti-PD-1 monoantigen CS1003 for the treatment of hepatocellular carcinoma.
- 4. Regency BioTrepri single resistance is a PD-1 monoantigen independently developed by Regency Bio, which was first approved in China at the end of 2018 for local progress or metastasis melanoma after the failure of previous standard treatments, and is the first Chinese-made PD-1 monoantigen to be approved for listing in China.
March, Ripley's monoantigeno-anti-acetinine treatment for mucosal melanoma was approved by the FDA for orphan drugs.
according to Junshi Bio News, Ripley monoanti is the first variety to be awarded "orphan drug eligibility" in the field of mucosal melanoma since the Orphan Drugs Act was enacted.
, Ripley monoantigen for the treatment of nasopharyngeal cancer and soft tissue sarcoma has been awarded orphan drug qualification by the FDA, so far, Ripley monoanti has been qualified for three orphan drugs.
5. Xinda Biosynthetics, a PD-1 monoclonal antibody developed in collaboration with Eli Lilly and Company, was approved for listing in China in December 2018 for the treatment of recurring/refractic classic Hodgkin's lymphoma and has been included in China's national health insurance list.
In addition, two new adaptive applications for non-scaly non-small cell lung cancer (NSCLC) first-line treatment, and two new adaptation applications for the treatment of first-line squamous NSCLC patients in the joint use of Cindili monoantigen and Gissitalbin have also been accepted by NMPA.
this year, Syndicated has been eligible for two FDA orphan drug treatments for T-cell lymphoma and esophageal cancer.
6. Baiji Shenzhou Tyrelli Pearl Monoanti is a humanized IgG4 monoclonal antibody against the immune checkpoint subject PD-1.
May this year, Retiliju single anti-treatment of stomach cancer, including gastroesophageal combined cancer was eligible for FDA orphan drugs.
in addition, Zanubrutinib, Baiji Shenzhou, was awarded three orphan drug qualifications in August this year for the treatment of lymphoma in the fringes of the lymph nodes, lymphoma in the spleen fringes and lymphomas in the outer edge of the mucous membrane-related lymph nodes.
Zebtini, an oral BTK small molecule inhibitor developed by Baiji Shenzhou, was approved in China in June for use in adult suite lymphoma patients who have received at least one treatment in the past and in adults with chronic lymphoblastic leukemia (CLL)/small lymphocytic lymphoma (SLL) who have received at least one treatment in the past.
Asaan Pharmaceuticals 4 products obtained 8 orphan drug certification in April this year, the U.S. FDA has awarded assassin Pharmaceutical Core in the research variety HQP1351 orphan drug qualification for the treatment of chronic myeloid leukemia (CML), which is ascension Pharmaceuticals obtained the first orphan drug qualification.
HQP1351 is a class 1 new drug developed by Asaan Pharmaceuticals, an oral third-generation BCR-ABL inhibitor.
, Aceh Pharmaceuticals has obtained orphan drug qualification for several products, and so far, Asheng Pharmaceuticals has obtained 8 FDA orphan drug certifications in the research of new drugs.
APG-115, an MDM2-p53 inhibitor, has conducted several clinical studies in China and the United States on the treatment of solid and blood tumors, and has shown considerable potential in preclinical studies for the treatment of stomach cancer.
at the moment.
has been qualified to treat soft tissue sarcoma, acute myeloid leukemia and stomach cancer.
APG-2575 is a new type of oral Bcl-2 selective small molecule inhibitor, is the first in China to enter the clinical stage, local enterprises developed Bcl-2 selective inhibitor.
year, APG-2575 was approved for multiple myeloma chronic lymphoblastic leukemia and Fahrenheit globulinemia.
APG-1252 selectively inhibits Bcl-2 and Bcl-xL protein repair apoptosis.
September, APG-1252 for the treatment of small cell lung cancer (SCLC) was eligible for orphan drugs.
APG-1252 is currently conducting clinical Phase I dose climbing trials in the United States and Australia for patients with advanced cancer, IB/II stage trials in the United States for patients with relapse recurring SCLC with combined yew alcohol, and single-drug Clinical Phase I dose climbing trials for SCLC patients in China.
addition to the above-mentioned products, Zezi Pharmaceuticals Jaktini tablets, Garcos JAB-3312, Gan Li Pharmaceuticals GLR2007, Dizhe Pharmaceuticals DZD4205, Yi Noi bio Y-2 tongue tablets, Yuan biological HY1003, China Resources Medicine NIP292 tablets, Dano Pharmaceutical TNP-2092 and many other new Chinese drugs have also been identified as orphan drugs in the United States.
orphan drugs have the characteristics of difficult research and development, high cost, small target market, poor profitability and so on, so many pharmaceutical companies are initially reluctant to enter the field.
, but under a series of support policies, orphan drugs have become one of the most profitable sectors in the pharmaceutical industry.
years, many multinational pharmaceutical giants have joined the ranks of orphan drug research and development, and has reaped heavy returns.
There are about 20 million rare disease patients in China, before 2020, China's research and development of "orphan drugs" is still in a blank, rare patients' treatment drugs basically rely on foreign imports, resulting in many rare disease patients can only choose expensive imported drugs or no drugs available.
, China's "orphan medicine" research and development has made a breakthrough, congratulations on these new drugs qualified as orphan drugs, hope that their global development is progressing smoothly, as soon as possible to benefit more patients.