A new study conducted by US National Institutes of Health scientists shows that Sanofi (France)'s drug Mozobil (plerixafor injection) may have promise in the treatment of patients with WHIM syndrome, a rare immune deficiency.
IHS Global Insight Perspective | |
Significance | Further clinical trials in that indication may contribute to expand Mozobil's label in the field of rare disease, an area where French firm Sanofi is aiming to foray after the acquisition of US-based Genzyme. In another development, its US subsidiary decided to return PTC Therapeutics' worldwide rights to ataluren. |
Implications | The restructuring of the deal is the result of a reassessment of Genzyme's pipeline. Genzyme retains an option to commercialise ataluren in indications other than nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) outside the United States and Canada. |
Outlook | With Genzyme, Sanofi boosts its chances of becoming a leader in the area of rare diseases. |
Plerixafor As Treatment for WHIM Syndrome
A new study conducted by scientists from the US National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), shows that Sanofi (France)'s FDA-approved drug Mozobil (plerixafor injection) may have promise in the treatment of patients with WHIM (warts, hypogammaglobulinemia, infections, myelokathexis) syndrome, a rare immune deficiency. Over a seven-day period, three adult patients with WHIM syndrome were given six injections of increasing doses of plerixafor. The NIAID team observed that the numbers of nearly all immune-cell deficiencies in the three patients increased to normal levels, with only minimal adverse side effects at the highest doses. In the United States, the drug is approved for use in patients undergoing a bone-marrow transplant.
Approximately 60 patients worldwide have been diagnosed with WHIM syndrome so far, according to the NIAID. The current treatment involves intravenous immunoglobulin and granulocyte colony-stimulating factor, but is difficult to administer, costly and partially effective.
Genzyme Returns Rights to Ataluren
In another development, Genzyme (US)—which originally developed plerixafor and is now part of the Sanofi group—decided to restructure its agreement with US-based PTC Therapeutics. Under the restructured agreement, PTC regains worldwide rights to ataluren while Genzyme retains an option to commercialise ataluren in indications other than nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) outside the US and Canada. The restructuring is the result of a reassessment of Genzyme's pipeline.
Under the initial agreement in 2008, Genzyme paid USD100 million in upfront cash for the right to co-develop and co-market ataluren. PTC loses a valuable partner which had promised double-digit royalties on sales of the product upon approval.
Outlook and Implications
The acquisition of Genzyme has anchored Sanofi in the area of rare disease and Genzyme is now reassessing its pipeline to refocus on the most promising projects. Genzyme's research and development pipeline is no doubt a windfall for Sanofi, which has lacked the strong compounds necessary to become less vulnerable to generic competition before acquiring the leader in the rare disease field. Positive effects observed with the use of Mozobil on patients with WHIM deficiency will now need to be proven within long-term studies in adults first, and then in children. Mozobil is one of the latest drugs launched by Genzyme before its takeover by Sanofi (see United States - France: 16 February 2011: Sanofi-Aventis Agrees to Purchase Genzyme for US$20.1 Bil.). Over the second quarter of 2011, Sanofi's sales grew 6.9% year-on-year to EUR8.3 billion (USD11.72 billion) thanks to Genzyme. Excluding the EUR796 million in sales brought in by Genzyme during the second quarter, sales fell by 4% year-on-year.