The year 2005 saw the approval of a number of orphan drugs, including Naglazyme (galsulfase), the first specific therapy approved for the treatment of mucopolysaccharidosis VI (MPS VI, also known as Maroteaux-Lamy syndrome). In 2006 watch for the approval of Shire plc’s Elaplase (idursulfase) as the first-ever treatment for mucopolysaccharidosis II (MPS II, known as Hunter syndrome). Elaplase was formerly known as iduronate-2-sulfatase (I2S) when it was being developed by Transkaryotic Therapies (TKT). When Shire acquired TKT in 2005 it continued developing the drug.
Shire submitted its application for Elaplase approval to the U.S. Food and Drug Administration {FDA} in November 2005. Shire requested a Priority Review, which would mean a response from the FDA in six months. Shire also submitted an application for Elaplase to the European Medicines Agency (EMEA) on December 1, 2005. The EMEA approval process usually takes 12 months, so Elaplase may get in under the wire before the end of 2006.
Another drug approval to watch for is a new indication for Novartis’ Glivec (imatinib, marketed in the U.S. as Gleevec). Glivec has previously received FDA and EMEA approval for treatment of chronic myeloid leukemia and gastrointestinal stromal tumors. Novartis submitted applications to both the FDA and EMEA in December 2005 for approval of Glivec in the treatment of adult patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). If approved for this new indication, Glivec would be the first therapy that specifically targets a genetic abnormality responsible for Ph+ ALL. Look for FDA approval in mid-2006 and EMEA approval by the end of the year.
Mergers and acquisitions follow biotech successes
Biotechnology companies both large and small have improved their cash flow with successful drug development in 2005. I’ve already mentioned Shire plc, a rapidly growing global specialty pharmaceutical company, and its acquisition of Transkaryotic Therapies (in April 2005). Novartis had success in 2005 with the approval of its orphan drug Exjade (desferasirox) and its breast cancer treatment Femara (letrozole). During 2005 Novartis acquired a stake in Chiron Corporation, a vaccine manufacturer. Novartis has received US regulatory approval to acquire the remaining 58% stake in Chiron, and expects to complete the acquisition of Chiron in the first half of 2006, subject to approval by a majority of Chiron's shareholders.
In addition, Novartis has decided to explore the benefits of acquiring the Swiss vaccines company Berna Biotech AG and combining its operations with those of Chiron Corporation. Although this is no guarantee that an offer for Berna will be made in 2006, the acquisition would complement Novartis’ current operations.
In June 2005 the world’s largest drug maker, Pfizer, received FDA approval, and in November 2005 EMEA approval, for Revatio (sildenafil citrate) for the treatment of pulmonary arterial hypertension, a rare blood vessel disorder of the lungs. Sildenafil citrate is the active ingredient in Viagra, Pfizer's big-selling erectile dysfunction medication. Pfizer acquired Idun Pharmaceuticals in February 2005 and Vicuron Pharmaceuticals in September 2005. Pfizer also made a $50 million investment in Perlegen Sciences in late December 2005, taking a twelve percent position in the private biotechnology company.
Outlook for 2006
This merging of companies large and small shows no signs of letting up in 2006. Let’s hope that one of the benefits of all the acquisitions taking place in biotech--the combining of all that research brainpower and resources--will be the development and approval of more orphan drugs and new ways to use existing drugs to treat rare conditions.
