Wanted: Orphan Drugs, Antivirals Dominate China's Latest Fast Track List
Executive Summary
Half of the newly selected imported drugs slated for fast track review in China are rare disease treatments, followed by antiviral drugs for influenza and HIV, reflecting key medical needs in the country.
Unlike previous rounds of fast track approval review designations in China, in which oncology drugs took the lead, this time orphan drugs are shining.
The latest list of selected products released March 28 by China’s National Medical Products Administration (NMPA) appears to have a clear message for pharma companies around the world– bring your approved orphan drugs to China, the sooner the better.
The agency’s new drug review subsidiary, the Center for Drug Evaluation, noted that the newly fast track-listed drugs are those with the largest unmet needs in China.
Among the total of 30 products, rare diseases treatments accounted for half. These included not only more established drugs but also new therapies such as Kyowa Hakko Kirin Co. Ltd.’s Crysvita (burosumab), which was approved last February by the European Medicines Agency for X-linked hypophosphatemia.
Other notables include Novo Nordisk AS’s insulin preparation Levemir (insulin detemir) for Noonan syndrome and Prader-Willi syndrome. Several enzyme replacement therapies are also listed including BioMarin Pharmaceutical Inc.’s Aldurazyme (laronidase) for MPS I, Shire PLC’s Elaprase (indursulfase) for MPS II and Genzyme Corp.’s Fabrazyme (agalsidase beta) for Fabry’s disease.
In effect, three drugs will be listed for Fabry’s disease in China, the other two being Shire’s Replagal (agalsidase alfa) and Amicus’s Galafold (migalastat).
Similarly, three pulmonary artery hypertension drugs are now set for priority reviews in China: Janssen-Cilag GMBH’s Tracleer (bosentan),
Pfizer Inc.’s Revatio (sildenafil) and Toray Industries Inc.’s Careload (beraprost sodium).
Other orphan drugs designated for priority review were Bioverativ Inc.’s Alprolix (coagulation Factor IX) for hemophilia B, Ruconest (recombinant human C1-inhibitor) from Pharming Group NV for hereditary angioedema, Sanofi’s Lemtrada (alemtuzumab) for multiple sclerosis, Radicava (edaravone) from Mitsubishi Tanabe Pharma Corp. for amyotrophic lateral sclerosis.
Out of the orphan drug companies, Sanofi/Genzyme and Takeda/Shire each scored two drugs in the new listing, and both have showed their intention of strengthening their rare disease product portfolios in China, an increasingly important market for such products. The most recent list is also another indication that the government is willing to introduce more and newer treatments to the market.
During the International Pharmaceutical Innovation Forum held March 26 in Beijing, Sanofi CEO Olivier Brandicourt told the audience that the French drug maker stands ready to help improve the treatment and diagnosis of rare conditions in China.
Other Focus Areas
Orphan drugs aside, antivirals are also emerging as a major area of unmet need in China, underscored by the new listing of four new therapies for fast track review.
Included were Gilead Sciences Inc.’s Biktarvy (bictegravir, emtricitabine and tenofovir alafenamide) for HIV, which was approved in February in the US, Shionogi & Co. Ltd.’s Xofluza (baloxavir marboxil) for seasonal flu A and B, Daiichi Sankyo Co. Ltd.’s Inavir (laninamivir octanoate hydrate) to treat or prevent flu, and AbbVie Inc.’s Maviret (glecaprevir/pibrentasvir) for hepatitis C viral infections.
Meanwhile, pediatric drugs are another area of focus in the listing, which included Increlex (mecasermin) for children with growth deficiency, and Santen Pharmaceutical Co. Ltd.’s Verkazia (ciclosporin) for children's eye infections, and Aucta Pharma's Vigadrone (vigabatrin) was selected in combination use to treat partial seizures in infants, young children and adults.
From the editors of PharmAsia News.