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In The News

BioMarin Rewrites Playbook for Small-Market Drug Approvals

Few people have heard of Batten disease, a rare genetic disorder that robs children of the ability to walk and talk before the first day of kindergarten and often kills them by the age of 12.

But when BioMarin Pharmaceutical Inc.unveiled early data this month on a mid-stage trial of its experimental Batten disease drug, people took notice. Families of young patients saw the promise of arresting a form of the deadly disease; Wall Street saw hope that the San Rafael company (NASDAQ: BMRN) could expand its portfolio of drugs targeting rare diseases that are tough to pronounce and tougher still to stop.

Indeed, early but strong data from BioMarin's study of BMN-190 in Batten disease, its $680 million acquisition this month of Prosensa Holding NV and that company's Duchenne muscular dystrophy drug, and the soaring prices of rare-disease drugs have helped push BioMarin's stock up 61 percent over the past seven months to a 52-week high near $100 per share.

Along the way, BioMarin is rewriting the playbook for how drugs are approved for rare diseases with fewer than 200,000 U.S. patients. The lead chapter focuses on Europe.

In BioMarin's study of BMN-190 in infantile neuronal ceroid lipofuscinosis — a form of Batten disease known as CLN2 — only three of the 24 patients are from the United States, for example. Most of the children receive an injection every other week at University Hamburg-Eppendorf in Germany or Bambino Gesù Children's Hospital in Rome.

Europe, said BioMarin's French-born president and CEO, Jean-Jacques Bienaimé, offers more flexibility in setting up a trial. That saves time and money, which is particularly important when the process of finding and enrolling patients comprises about 40 percent of the cost of many drug trials.

In a rare disease like Batten, which is commonly misdiagnosed or identified too late for a child to join a clinical trial, those costs can run even higher.

There are only 400 to 600 patients diagnosed worldwide with the CLN2 form of Batten.

BioMarin is familiar with the European routine. The first clinical trial for its most recently approved drug — Vimizim, for a skeleton and muscle deforming condition known as Mucopolysaccharidosis type IVA — occurred in Europe.

Vimizim was approved in the United States in February 2014 and in Europe two months later. Its abbreviated 2014 sales are expected to be $75 million to $77 million, thanks in large part to its $380,000-a-year price.

In fact, those six-figure prices for extending patients lives by years are a big reason why investors love BioMarin. Drisapersenthe late-stage Duchenne muscular dystrophy drug BioMarin picked up in its acquisition of Prosensa, could cost in the $300,000-a-year range.

Drisapersen also could get a European boost. Although the drug failed a late-stage trial led by GlaxoSmithKline, Bienaimé said European regulators could be more open to approving drisapersen based on the totality of the drug's clinical data.

BioMarin, which has the FDA's "breakthrough therapy" designation for drisapersen and is racing Sarepta Therapeutics Inc. of Cambridge, Mass., to win the first U.S. Duchenne muscular dystrophy drug approval, expects this quarter to ask the FDA to approve the drug. It will file for European approval in the second quarter.

BMN-190, a recombinant protein, could soon follow if it is as effective as that demonstrated in the first nine patients treated for more than six months. Of those nine, Bienaimé said at this month's J.P. Morgan Healthcare Conference, a walk-talk test found no decline in six of them, which could mean that the drug stabilizes the disease.

The remaining three patients' walk-talk scores, Bienaimé added, actually improved.

The drug is delivered through a port in the skull that is connected to a catheter that leads to a child's brain.

BioMarin started the trial in September 2013.

"The early results make us feel like we're headed in the right direction," said Margie Frazier, executive director of the Columbus, Ohio-based Batten Disease Support & Research Association.

The association worked with BioMarin to enroll some of the patients in the study. It also is supporting the families of patients undergoing treatment at Nationwide Children's Hospital in Columbus.

BioMarin hopes to expand access to BMN-190 to siblings of Batten disease patients who have the disease but haven't yet shown symptoms. The addition of those children could demonstrate if the drug actually blocks the disease, Bienaimé said, as well as halts its progression.

If BMN-190's data continues to impress, Bienaimé said, U.S. and European regulators could cut to the chase and not wait for a full year of data from all 24 patients, which will come by the end of this year. Or regulators may tell BioMarin to add more patients to the study.

Regardless, the tight-knit Batten disease community of patients, families, caregivers and researchers will be watching closely, said the association's Frazier.

"All of the families, especially the newly diagnosed patients, they're wide awake to what is happening in this study," she said.

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