Ceprotin, Takeda's rare dz, gives 11 yrs for reexamination

Korea's first approved treatment for severe congenital protein C deficiency

The Central Pharmacist Review Board said, Insufficient patients, It's hard to evaluate in 4-6 years

On the 2nd, Takeda's Ceprotin, which was first approved as a treatment for severe congenital protein C deficiency in Korea, was given an 11-year review period after marketing.

 

Severe congenital protein C deficiency is a rare genetic disease that causes fatal defects in blood clot control due to a lack of protein C, a type of vitamin K-dependent anticoagulant, and is defined when protein C levels are less than 1% (less than 1 IU/dL), and the incidence rate is estimated to be 1 in 4 million newborns.

 

Ceprotin is the first human protein C drug approved for patients with severe congenital C protein deficiency and was approved for "prevention and treatment of venous thrombosis and fulminant purple hemiplegia in patients with severe congenital protein C deficiency in children and adults." Prior to Ceprotin's approval, the Central Pharmaceutical Review Committee's Pharmaceutical Affairs Subcommittee-Rare Medicine Subcommittee discussed the validity of the re-examination period when licensing biopharmaceutical items.

 

On this day, the committee members decided that it was appropriate to set a period under the Rare Disease Control Act in consideration of the prevalence rate as an item prescribed for rare diseases.

 

It also said that 11 years of re-examination, including pediatric indicators, should be given to secure sufficient safety information.

 

It is reasonable to grant a 10-year re-examination period, including a pediatric indication, and a 10-year re-examination period, which was consulted by the central pharmacy, considering the pharmacological law, the re-examination system, and the characteristics of rare diseases.

 

"It is difficult to judge that there is no appropriate treatment for severe congenital protein C deficiency because there are standard treatments," a member said.

 

"However, it is confirmed that there are limitations on standard treatments and risks to be applied to children." The opinion is that it is difficult to evaluate post-marketing safety due to a lack of patients by granting a 4-6 year review period for congenital genetic diseases, which have symptoms mainly in the early stages of newborns and have a very high mortality rate from complications.

 

Considering the nature of special diseases and the characteristics of the domestic medical environment, a total of 11 years of re-examination period, including pediatric diseases, will be given.