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BioMarin's (BMRN) Brineura Review Period Extended by FDA
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BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that the FDA has extended the review period of its biologics license application (BLA) for Brineura (cerliponase alfa) by three months. The company is looking to get Brineura approved for the treatment of children with CLN2 disease, a form of Batten disease.
What Led to the Delay?
BioMarin announced that the FDA had requested an updated efficacy data cut from the ongoing extension study during its initial review of the BLA. The company has submitted additional data as part of its BLA for Brineura and the FDA has designated this submission as a major amendment to the application. Owing to this, the regulatory body will now announce its decision by Apr 27, 2017, instead of the previous date of Jan 27, 2017.
Approximately 8 months of additional data (81-week data) continued to show that motor-language scores of the majority of patients, when treated with Brineura, were stable, representing a considerable reduction in disease progression, compared to natural history. The additional data were consistent with the previously submitted 48-week data, thereby showing durable and consistent treatment response with Brineura.
However, in its press release, BioMarin stated that the FDA has not revealed its basis for designating this submission as a major amendment. The regulatory agency plans to hold an advisory committee meeting in the future.
We note that the company has submitted a marketing authorization application to the European Medicines Agency (EMA) for Brineura. The filing is presently undergoing validation, with an update expected shortly. The EMA has granted BioMarin's request for accelerated assessment, which will shorten the candidate’s review period.
However, the latest development on the regulatory front is disappointing. Per BioMarin’s press release, CLN2 disease is a rapidly progressing, fatal neurodegenerative disease that impairs the ability to walk and talk in the majority of affected children by approximately six years of age.
The company estimates CLN2 disease to affect approximately one in 200,000, with nearly 1,200 to 1,600 children in the company’s commercial territories.
Considering that there is no approved product to prevent, stop, or reverse this disease, a timely approval will be a huge boost for the company. We note that Brineura enjoys Orphan Drug status in both the U.S. and the EU.
Meanwhile, BioMarin has implemented an early access (compassionate use) program as planned to offer Brineura to additional CLN2 patients, before gaining approval. This program, which is being conducted under a protocol, began last month.
BioMarin carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Anika Therapeutics Inc. (ANIK - Free Report) , Geron Corporation (GERN - Free Report) and Corcept Therapeutics Incorporated (CORT - Free Report) . All the three stocks sport a Zacks Rank #1 (Strong Buy).
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BioMarin's (BMRN) Brineura Review Period Extended by FDA
BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that the FDA has extended the review period of its biologics license application (BLA) for Brineura (cerliponase alfa) by three months. The company is looking to get Brineura approved for the treatment of children with CLN2 disease, a form of Batten disease.
What Led to the Delay?
BioMarin announced that the FDA had requested an updated efficacy data cut from the ongoing extension study during its initial review of the BLA. The company has submitted additional data as part of its BLA for Brineura and the FDA has designated this submission as a major amendment to the application. Owing to this, the regulatory body will now announce its decision by Apr 27, 2017, instead of the previous date of Jan 27, 2017.
Approximately 8 months of additional data (81-week data) continued to show that motor-language scores of the majority of patients, when treated with Brineura, were stable, representing a considerable reduction in disease progression, compared to natural history. The additional data were consistent with the previously submitted 48-week data, thereby showing durable and consistent treatment response with Brineura.
However, in its press release, BioMarin stated that the FDA has not revealed its basis for designating this submission as a major amendment. The regulatory agency plans to hold an advisory committee meeting in the future.
We note that the company has submitted a marketing authorization application to the European Medicines Agency (EMA) for Brineura. The filing is presently undergoing validation, with an update expected shortly. The EMA has granted BioMarin's request for accelerated assessment, which will shorten the candidate’s review period.
However, the latest development on the regulatory front is disappointing. Per BioMarin’s press release, CLN2 disease is a rapidly progressing, fatal neurodegenerative disease that impairs the ability to walk and talk in the majority of affected children by approximately six years of age.
The company estimates CLN2 disease to affect approximately one in 200,000, with nearly 1,200 to 1,600 children in the company’s commercial territories.
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Considering that there is no approved product to prevent, stop, or reverse this disease, a timely approval will be a huge boost for the company. We note that Brineura enjoys Orphan Drug status in both the U.S. and the EU.
Meanwhile, BioMarin has implemented an early access (compassionate use) program as planned to offer Brineura to additional CLN2 patients, before gaining approval. This program, which is being conducted under a protocol, began last month.
BioMarin carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Anika Therapeutics Inc. (ANIK - Free Report) , Geron Corporation (GERN - Free Report) and Corcept Therapeutics Incorporated (CORT - Free Report) . All the three stocks sport a Zacks Rank #1 (Strong Buy).
Confidential from Zacks
Beyond this Analyst Blog, would you like to see Zacks' best recommendations that are not available to the public? Our Executive VP, Steve Reitmeister, knows when key trades are about to be triggered and which of our experts has the hottest hand. Click to see them now>>