New drugs that have received conditional approval of marketing in China

Osimertinib Tablets (Tagrisso)

Osimertinib is an oral third-generation EGFR-TKI (epidermal growth factor receptor tyrosine kinase inhibitor) developed by AstraZeneca that can effectively act on EGFRm (TKI sensitivity associated mutation) and T790M-positive (TKI resistance mutation) EGFR, thus to overcome the T790M resistance of the first-generation and second-generation EGFR-TKIs, and become the choice of patients with T790M-mutant NSCLC (non-small cell lung cancer) after failing first-line treatment.

Osimertinib has early received the breakthrough therapy and orphan drug designations of FDA, and received the accelerated approval of FDA on November 13, 2015; AstraZeneca filed the imported drug registration application of the pharmaceutical product to CFDA in August 2016, and received the conditional approval of marketing of CFDA on March 24, 2017 after the drug was included in the priority review, which would be used for the treatment of adult patients with EGFR T790M mutation-positive locally advanced or metastatic NSCLC, as detected by test, whose disease has progressed on or after EGFR-TKI therapy.

The main basis to support the conditional approval of this drug is the global pivotal Phase II studies AURA extension and AURA2, with the primary endpoint of ORR; and the results showed that ORR (N=411) was 66.1%, DCR was 91%, and median PFS was 9.7 months; and a Phase I PK study and Phase II clinical study conducted in China proved that Chinese patients were similar with global patients in terms of PK characteristics, and the exposure was not affected by the race.

Human Papillomavirus 9-valent Vaccine (Gardasil 9)

Human papillomavirus (HPV) vaccine is the world’s first vaccine product indicated for cancer; developed by MSD, it can induce the human body’s immunity by using the special protein coat on the virus, to prevent occurrence of cancer associated with HPV infection.

MSD filed the imported drug marketing application for Human Papillomavirus 9-valent Vaccine (9vHPV) to CDE in April 2018; and it only took CFDA 8 days to conditionally approve the marketing application of 9vHPV on April 28, 2018, which would be used in girls and women 16 through 26 years of age for the prevention of cervical, vulvar, vaginal, and anal cancers, genital warts, persistent infection, precancerous lesions or atypical lesions caused by HPV. This conditional approval is based on the approval data of the previous bivalent and tetravalent HPV vaccines, completely adopts the overseas clinical trial data in the world, and requires further expanding the clinical study and pharmaceutical study of the Chinese population after the marketing.

FDA has approved 9vHPV for females and males aged 9-26, however, this conditional approval in China only approves it for people aged 16-26, because the overseas data include only results of the immune-bridging trial of vaccinated people aged 9-15, which contained limited clinical data of East Asian Chinese people, and showed serious adverse reactions. The age of people to receive the vaccine will be decided whether to be expanded subsequently according to the clinical study results in China.

Alglucosidase Alfa for Injection (Myozyme)

Alglucosidase Alfa for Injection, with the trade name: Myozyme, is a drug for the treatment of Pompeii disease (acid alpha-glucosidase deficiency), developed by Genzyme under Sanofi, and is also world’s first and only drug for Pompeii disease, acting through enzyme replacement therapy to replace the acid alpha-glucosidase that is not existing or is deficient. Myozyme was first approved by FDA in 2006, and was approved by FDA in May 2010 for the treatment of patients 8 years and older with late onset Pompeii disease; its indication was expanded in 2014 as it was approved by FDA for all Pompeii patients of any age or phenotype.

Sanofi filed the imported drug marketing application with exemption of Phase III clinical trial of the variety to CFDA in June 2015. CFDA agreed the conditional approval of marketing of the drug on November 4, 2015 as CDE considered based on the completed clinical trial on the safety and effectiveness that enrolled 80 patients with infantile onset Pompeii disease and 101 patients with late onset Pompeii disease as well as 5 clinical studies underway at that time, with reference to the drug use experience of the product after marketing in the U.S., Europe, and Japan, etc., and considered the particularity of the associated rare disease, however, the drug’s effectiveness, safety, and dosage, etc. for the Chinese patients should be further assessed after the marketing, with the number of cases of not less than 50, and clinical medication duration of at least 1 year.

Erxiao, Master of Pharmacy, pharmaceutical industry practitioner, engaged in imported drug and new drug registration, and focused on registration regulations and new drug dynamics.

Read More: What is "Conditional Approval"

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