Content about Rare diseases

LEXINGTON, Mass. — Drug maker Shire has started a research program focused on rare diseases and their effects on patients and healthcare professionals, the company said Monday.

Shire announced the launch of the Shire Rare Disease Impact Report, which includes surveys of patients, caregivers, physicians, payers and others in the United States and United Kingdom, with results expected in the first half of 2013.

CAMBRIDGE, Mass. — An experimental drug for treating a rare genetic disease appears effective, according to a late-stage clinical trial.

NEEDHAM, Mass. — In the battle to find a cure for amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, scientists from Belgium have made a rare discovery: a new gene that influences survival time of ALS. The findings, reported in a recent issue of Nature Medicine, were funded in part by CVS/pharmacy and the ALS Therapy Alliance, a Boston-based nonprofit. The findings also confirm another recent study that identified the same pathway to finding a treatment for ALS.

NEW YORK — Drug makers could strike big with new treatments for rare diseases, according to a new report by a market research firm.

LEXINGTON, Mass. — British drug maker Shire is looking to make a biotech drug for a rare genetic disorder at a new manufacturing plant in Massachusetts.

The company said Tuesday that it had filed with the Food and Drug Administration and the European Medicines Agency for approval to produce the Gaucher disease drug Vpriv (velaglucerase alfa) at its Lexington, Mass., plant. The company expects to win approval to make the drug there early next year.

WOONSOCKET, R.I. — CVS/pharmacy kicked off on June 5 its 10th annual in-store "Researching a Cure" fundraising campaign to support the fight against amyotrophic lateral sclerosis.

Funds raised through the campaign benefit the ALS Therapy Alliance, an organization dedicated to advancing ALS research. Through the Researching a Cure campaign, CVS/pharmacy has raised more than $23 million in its stores and online to fund new clinical research with the aim of discovering a treatment or cure for ALS. The 2011 campaign will run through June 25.

SAN DIEGO — Pfizer is partnering with San Diego biotech company Zacharon Pharmaceuticals to develop treatments for rare genetic disorders Zacharon said Thursday.

Under the collaboration, which could garner Zacharon up to $210 million, the companies will discover and develop pharmaceutical drugs for treating lysosomal storage disorders, a class of illnesses that includes Fabry disease, Gaucher disease, Pompe disease and Tay-Sachs disease.

CAMBRIDGE, Mass. — It appears that French drug maker Sanofi-Aventis’ efforts to acquire U.S. biotech company Genzyme are getting somewhere, according to company and media reports.

CRANBURY, N.J. British drug maker GlaxoSmithKline will work with U.S.-based Amicus Therapeutics to develop a drug for a rare genetic disease.

The two companies announced a deal to develop and commercialize Amigal (migalastat hydrochloride), a treatment for Fabry disease. Under the deal, GSK will pay Amicus $30 million upfront, as well as milestone payments of up to $170 million and royalties on future sales.

MUMBAI, India The Food and Drug Administration has approved a generic version of a drug for a devastating muscular disorder.

Sun Pharmaceutical Industries announced Tuesday the approval of riluzole hydrochloride in the 50-mg strength. The tablets are used to treat amyotrophic lateral sclerosis or ALS, also known as Lou Gehrig’s disease.

The drug is a generic version of Sanofi-Aventis’ Rilutek, which has sales of around $50 million, according to Sun.

NEW YORK Biotech drug maker Genzyme’s takeover by French drug maker Sanofi-Aventis may have run into trouble, according to published reports.

Bloomberg reported Wednesday that Sanofi was unwilling to raise its offer of $70 per share for Genzyme, a Cambridge, Mass.-based company that has carved a niche for itself by manufacturing treatments for such rare genetic disorders as Fabry disease and Pompe disease but lately has been dogged by manufacturing problems.