BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) has released data from its 48-week phase1/2 pivotal study, for cerliponase alfa. The drug is being developed as a treatment for CLN2, a form of Batten disease. The disease is known to be fatal and is a neurodegenerative disease, with no approved treatments. Added to this, CLN2 also causes children to lose their ability to walk and talk, by the age of 6.

As per the details of the findings, efficacy end-point for the drug was approximately 80% lower than expected in the untreated population. However, the studies also reported that a total of 65% of patients were able to experience stabilization of the disease. The drug had been administered through ICV infusion, on a weekly basis. The company expects that approximately 1,200 to 1,600 children, in BMRN’s commercial territories alone, suffer from CLN2.

The company made use of the standardized motor and language score, in CLN2 patients, with 0 indicating no function and 3 indicating normal function, to assess their results. The patients had entered the trial, with an average score of 2.1, which was successfully reduced to 0.43, at the end of the 48-weeks, in 21 patients. However, two patients, who had entered with a score of 6, remained unchanged.

Added to this, the FDA has also granted BioMarin’s BMN 270 an orphan drug rating. The drug is mainly used to treat hemophilia A. The drug is currently undergoing a phase 1/2 study, to determine the safety of the drug in 12-patients. The company would also provide an update on the program in April this year.

BioMarin is expected to file for approval of the drug, for treatmen of CLN2, by the middle of the year. The company is also expected to conduct a research meeting in April, to update investors on its pipeline of drugs for rare diseases.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) completed the March 2 trading session, with a loss of 0.15% to its share value, to reach a close at $87.44.