TOP12 global rare disease drugs in 2021: K drug sales of US$17.18 billion, BMS two blockbuster drugs ranked in the top three

Orphan drugs are drugs used to diagnose, prevent and treat rare diseas.

About 80% of rare diseases are inherited and mainly affect childr.

At present, oncology orphan drugs still occupy the majority of the market share of rare disease dru.

01Keytruda

Keytruda has been in the spotlight since it was approved for marketing in 201 This blockbuster tumor immunotherapy has shown promising efficacy in a variety of cancers and can be comprehensively used for the treatment of melanoma, head and neck cancer, non-small cell lung cancer, classic Hodgkin lymphoma, bladder cancer, gastric cancer,e.

02Revlimid

Revlimid is a blockbuster immunomodulator obtained after Bristol-Myers Squibb acquired Celge.

03Opdivo

As the first PD-1 cancer immunotherapy drug approved for marketing in China, the suggested retail price of Opdivo (nivolumab injection) is 9,260 yuan for 100mg/10mL, and 4,591 yuan for 40mg/4.

 04Trikafta

In October 2019, the.

 05Imbruvica

Imbruvica is an oral Bruton's tyrosine kinase (BTK) inhibitor jointly developed and commercialized by Pharmacyclics, an AbbVie company, and Janssen Biotechnology, a Johnson & Johnson compa.

06Hemlibra

In 2017, Hemlibra (emicizumab-kxwh) was approved by the US FDA for the routine prevention, prevention or reduction of bleeding events in adults and children with hemophilia A with factor VIII inhibito.

07Lynparza

Lynparza is the first PARP inhibitor to be marketed globally and was first approved by the US FDA in December 201 AstraZeneca and Merck reached a global strategic cooperation in oncology in July 2017 to jointly develop and commercialize Lynparza and another MEK inhibitor selumetinib for the treatment of various types of tumo.

 08Vyndaqel

Pfizer’s layout in rare disease treatment is also relatively early, with the main orphan drug Vyndaqel (tafamidis) for the treatment of transthyretin-related familial amyloid polyneuropathy (ATT.

 09Darzalex

Darzalex is the first approved fully human monoclonal antibody targeting CD3 It has a unique and innovative therapeutic mechanism and can directly bind to CD38, an important immunotherapy target on the surface of myeloma cells, and induce bone marrow through multiple mechanis.

10Soliris

Alexion's Soliris was approved by the FDA in March 2007 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults and children, and was subsequently approved for atypical hemolytic uremic syndro.

 11Venclexta

Currently, Venetoclax has been approved in more than 80 countries around the world for the treatment of chronic lymphocytic leukemia (CLL), small cell lymphoma (SLL), and acute myeloid leukemia (AM.

12Jakafi

In late 2011, Jakafi became the first JAK inhibitor approved by the FDA for the treatment of the bone marrow disease myelofibros.