China's Rare Disease Blockbuster Tafamidis Meglumine Soft Capsules, with Global Sales Exceeding USD 3.3 Billion, Sees Two Domestic Approvals

　　【Pharmaceutical Network Industry DynamicsOn June 18, according to the National Medical Products Administration, the chlorbenzoxamine soft capsule, a Category 4 generic drug applied for by Qilu Pharmaceutical, was approved for production, marking it as China's first domestically produced and top-rated generic. On June 25, the chlorbenzoxamine soft capsule from Nanjing Chia Tai Tianqing Pharmaceutical was also approved, becoming the second domestically produced version in China.
 

Tafamidis, developed by Pfizer, is a rare disease drug for treating adult patients with stage I symptoms of transthyretin amyloidosis polyneuropathy (ATTR-PN) and can effectively delay peripheral nerve damage. Currently, Pfizer's tafamidis series mainly includes Tafamidis Meglumine Soft Capsules and Tafamidis Soft Capsules.
 

The annual sales of Pfizer's Chlorbenzazole series have been increasing year by year after its launch. Especially with the increase in the number of approved countries, the global sales of this series continue to rise. Financial report data shows that the global sales of Pfizer's Chlorbenzazole series exceeded 2 billion US dollars in 2021 and surpassed 3.3 billion US dollars (more than 23 billion yuan) in 2023.
 

It is reported that Pfizer's Tafamidis Meglumine Soft Capsules were approved for marketing in China in February 2020. In October of the same year, its Tafamidis Soft Capsules were also approved in China for the treatment of adult wild-type or hereditary ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalizations.
 

Data shows that in 2022-2023, the sales growth rates of Pfizer's chlorbenzazole series products at the terminals of urban public hospitals, county-level public hospitals, urban community centers, and township health centers (referred to as Chinese public medical institutions) were 5831.12% and 118.97%, respectively, indicating significant market potential. However, industry insiders stated that with the approval and market entry of Qilu Pharmaceutical's and Zhengdatianqing’s chlorbenzazole meglumine soft capsules, Pfizer's key product may face market erosion in China.
 

Data show that ATTR-PN is a rare autosomal dominant hereditary amyloidosis caused by gene mutations, often affecting multiple systems in the body. The main clinical manifestations are symmetrical neurological dysfunction that progresses from the distal to the proximal limbs, involving motor, sensory, and autonomic nerves, and possibly accompanied by damage to other organs such as the heart, kidneys, and eyes.
 

The active ingredient in Chlorbenzazole Ammonium Soft Capsules, chlorbenzazole ammonium, is a transthyretin (TTR)StabilizerBy selectively binding to TTR, stabilizing TTR, and slowing down its dissociation into monomers, the production and deposition of ATTR amyloidosis are delayed, thereby achieving a therapeutic effect.
 

Industry insiders say that the listing of Qilu Pharmaceutical and Zhengda Tianqing's Tafamidis Methylglucamine Soft Capsules brings greater treatment hope for ATTR-PN patients. As Qilu Pharmaceutical stated in its announcement, with the improvement of drug accessibility, an increasing number of patients will receive more standardized treatment, thereby slowing disease progression, improving quality of life, and extending life expectancy.
 

It is reported that, in addition to Qilu Pharmaceutical and Zhengda Tianqing, Shijiazhuang Kerene Pharmaceutical, a subsidiary of Renhe Yikang, also completed the human bioequivalence (BE) trial of Clorbenzazole Pamoate Soft Capsules in April 2024. If the trial results are without issues, they may also apply for the product's market approval at a suitable time.
 

In addition, industry insiders said that as the generic versions of Tafamidis Meglumine Soft Capsules are successively approved for marketing in China, the financial burden on patients will also be reduced. It is reported that Tafamidis Meglumine Soft Capsules have not yet been included in the National Medical Insurance Drug List in China. As domestic generic drugs are approved for marketing, companies may actively participate in this year's adjustment of the National Medical Insurance Drug List to change the situation where the treatment cost of this drug remains high.
 

In terms of price, data shows that in August 2021, the price of Pfizer's Tafamidis Soft Capsules (61mg*30 capsules) dropped from 64,100 yuan/box to 24,650 yuan/box, preparing in advance for inclusion in the national medical insurance drug list that year; In December of the same year, the 2021 edition of China’s national medical insurance drug list officially included Tafamidis Soft Capsules. After renewal negotiations, the current price of Pfizer's Tafamidis Soft Capsules (61mg*30 capsules) has further decreased to 16,260 yuan/box.
 

Disclaimer: In no event shall the information or opinions expressed in this article constitute investment advice to any person.