Tuesday 13 March 2012, Amsterdam
This study estimates that the global Pompe disease therapeutics market grew at a Compound Annual Growth Rate (CAGR) of 57.1% during 2006-2011 and was worth $453.7m in 2011. The market is expected to record a CAGR of 13.8% from 2011 - 2019, to reach a value $ 1275.5m by 2019. Currently, Enzyme Replacement Therapy (ERT) is the major available therapy for Pompe disease patients. The ERT class includes Myozyme (alglucosidase alfa) and Lumizyme (alglucosidase alfa), both products are made by Genzyme (a Sanofi company) as approved drugs. Myozyme has been marketed in Europe and in the US from 2006, and in Japan from 2007. Myozyme has also received marketing approval in 44 countries. Myozyme is approved in US for treatment of early stage Pompe disease. Lumizyme has only been approved in the US region from 2010. Lumizyme is approved for the treatment of the late stages of Pompe disease.
The analysis indicates that the growth of the Pompe disease therapeutics market in the current period was attributed to an increase in awareness and approval of Lumizyme for sub-classes of Pompe disease. In addition, various foundations, organizations and support groups were active in educating and supporting Pompe patients. GlobalData also predicts that the Pompe disease therapeutics market will show as high a market growth rate in the forecast period due to increases in awareness levels and diagnostic rates.
Significant Unmet Need in Pompe Disease Therapeutics Market
The analysis indicates that the Pompe disease therapeutics market has significant unmet need, not only in terms of the limited number of therapeutic options available for treatment of the late stages of Pompe disease, but also a lack of safe treatment options for early-stage Pompe disease. Although ERT is approved for the treatment of Pompe disease, there are physician concerns about safety, efficacy and tolerability. The lack of an early diagnosis may lead to further disease progression and most of the early stage patients do not live more than a year. The annual cost of therapy with Myozyme exceeds $116,000m and so it is likely that many patients do not have access to medication. Alongside this, the symptoms of Pompe disease are unspecific and thus difficult to diagnose accurately.
The Current Competition in the Pompe Disease Therapeutics Market Is Weak
The current competition in the Pompe disease therapeutics market is particularly weak, and the market is only served by the two ERT drugs available in the market are Myozyme (alglucosidase alfa) for infants and Lumizyme (alglucosidase alfa) for adults... Both the marketed drugs are approved for different sub-classes of Pompe disease. These currently available options are moderately safe and efficacious and do not provide cost-effective treatment.
The market also witnesses weak competition because of some government regulations, such as orphan drug exclusivity.
Weak Pipeline with Lack of Novel Mechanisms of Action
The Pompe disease drug pipeline contains few products which could create new options for patients. Duvoglustat hydrochloride is the only first-in-class molecule that is in Phase II clinical trials which has a novel mechanism of action compared to the present therapies. Unlike others, Duvoglustat is using a pharmacological chaperone (a small molecule which selectively binds to proteins in cells and improves stability) approach which binds to the mutant enzyme and confers stability and improves trafficking, which in turn improves enzyme activity. The other first-in-class therapy includes rAAV1-CMV-GAA which uses gene therapy as the base mechanism. With the help of the AVV vector the drug supplies genes to produce deficient enzyme and improves the potency of the treatment. BMN 701 is a binder of mannose-6 phosphate receptor which increases ability to bind and trafficked enzyme towards lysosomes thus improve efficacy of treatment. Neo-GAA is a supply gene that delivers deficient enzymes and improves the potency of the treatment.