Rare Disease Report

Biomarin CEO Talks About Their Pipeline

JANUARY 11, 2013
RDR Staff

CEO of Biomarin, Jean-Jacques Bienaime, recently spoke at the 31st Annual J.P. Morgan Healthcare Conference. While Biomarin has 4 orphan drugs on the market that bring in approximately $500 million in revenue (Naglazyme® (galsulfase) for MPS VI, Aldurazyme® (laronidase) for MPS I, Kuvan® (sapropterin dihydrochloride) Tablets for PKU, and Firdapse (amifampridine) for LEMS), Mr. Bienaime focused most of his presentation on products they have in development, including Vimizin and Peg-Pal.

Vimizim (GALNS)

 Vimizim is an enzyme replacement therapy for patients with MPS IVA (Morquio Syndrome).  Morquio syndrome is an inherited lysosomal storage disorder in which patients are missing the enzyme N-acetyl-galactosamine-6-sulfatase. The net result is an accumulation of glycosaminoglycans throughout the body and they have features common to many MPS patients (course facial features, abnormal bone development, short stature) as well as  a plethora of other problems as gycosaminoglycans accumulate in various organs. short stature.

According to Mr. Bienaime, there are an estimated 3000 patients with Morquio syndrome worldwide and the biggest challenge is locating those patients. To date, Biomarin has identified 1200 patients.

vimizim 6mwt  337 225.jpgThe results from their recent pivotal trial showed Vimizim to achieve its primary endpoint (6 minute walk test)  and Biomarin plans to begin market authorization applications in the first quarter of 2013 in the United States. The ‘MOR-004’ study has not been published yet but Mr. Bienaime did provide some highlights from that study. It was a randomized trial in which 176 patients received placebo, 2 mg/kg/week of Vimizim, or 2 mg/kg/every other week of Vimizin. The study found that the 2 mg/kg/week dose significantly increased the 6 minute walking distance by 22.5 meters (P < 0.017). Improvements were also observed in secondary outcomes such as the 3 minute stair climb (increased) and urinary keratin sulfate levels (decreased). Mr. Bienaime also noted that the drug was well tolerated with most common adverse events being vomiting, pyrexia, headache, nausea, and cough – and many of those adverse events were also observed in the placebo group. Mr. Bienaime said that the results of that study will be presented in detail at the WORLD symposium next month in Orlando.

From a regulatory perspective, Mr. Bienaime stated that Biomarin plans to start market applications filings shortly with the United States and if the FDA grants priority review and meets PDUFA requirements, the first approval could be as early as the 4th quarter of 2013.  Mr. Bienaime also noted that ancillary studies are currently underway to examine the efficacy of Vimizim in patients under 5 yrs and in patients with limited ambulation. A cardiovascular study is also underway. All three studies are expected to be completed mid 2013.


PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase or ‘PAL’) is an enzyme substitute therapy for patients with phenylketonuria (PKU) which is an inherited metabolic disorder caused by a deficiency in the enzyme phenylalanine hydroxylase (PAH).  There are approximately 50,000 people with PKU worldwide. Patients with PKU build up toxic levels of the amino acid phenylalanine or "Phe" that can lead to numerous mental and psychological problems.  Currently, Biomarin has one product, Kuvan, to treat patients with PKU. Unfortunately, some patients do not respond to Kuvan as well as needed and Biomarin is hoping that Peg-Pal may be an alternative for those patients.

Mr. Bienaime noted that they have recently completed a phase 2 study with Peg-Pal and the reulst are promising. Of the 25 patients who were treated with Peg-Pal for one year, blood Phe levels were 68% lower than baseline and all patients met the NIH guidelines for Phe reduction in the study. To point out how phenomenal this data appears, Mr. Bienaime noted that their other drug, Kuvan, had only a 29% reduction in Phe levels and only 20% of patients were considered ‘responders’.

Other Drugs in Pipeline

Mr. Bienaime noted several other drugs currently in phase 1 or 2 trials. These included BMN-701 to treat Pompe disease, BMN-673 to treat solid tumors, and BMN-111 to treat Achondroplasia.

In addition to those drugs, Mr. Bienaime noted that Biomarin just announced the acquisition of Zacharon Pharmaceuticals. Mr. Beinaime stated that this acquisition will strengthen biomarin competencies in developing new treatments for lysosomal storage disorders such as MPS III, Tay Sachs, and Sandhoff.  

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