The global neurometabolic disorders market is expected to grow at a lucrative rate during the forecast period owing to the increasing incidence of various neurometabolic disorders. The growing demand for rapid diagnostic techniques over current techniques due to factors such as long turnaround time, supportive government funding, increased awareness amongst patients & the physicians are expected to be high impact rendering drivers for the market growth. On the contrary presences of the limited patient population to find the optimal dosage for the cure of the disease coupled with the high expenditure in the bioinformatics based research tends to restrain the market growth as the development of novel drugs is hindered.
The neurometabolic disorders market is categorized on the basis of type and route of administration. The types segment includes Gaucher's disease, Fabry disease, Pompe disease; mucopolysaccharidosis VI, and Niemann-pick type c disease. Out of the above-enlisted disorders, the Gaucher's disease held a substantial position in the market in 2014. The high prevalence of this disorder as compared to the other aforementioned disease has led to the increased demand for treatment of the disease and has contributed to the growth of the segment. Fabry disease is expected to be the fastest-growing segment over the forecast period owing to an increase in regulatory assistance and patient awareness. There are various pipeline products at different stages of development ranging from pre-registration to discovery and are to be developed over the forecast period. These are the drugs involved in the treatment of Fabry disease, with the comparative analysis involving the therapeutics assessment by drug target, route of administration, mechanism of action, and the type of molecule. One of the underlying reasons for the growth in the market of the Fabry disease is the special provisions for orphan drugs. On the basis of the route of administration, the neurometabolic disorders are categorized into oral and parenteral. The oral route of administration held the largest share in 2014 pertaining to the ease it provides to the patients. Parenteral route of administration is however less preferred by the patients owing to the pain at the site of delivery.
Geographically, the market is segmented into North America, Europe, Asia Pacific, Latin America, and MEA. North America held substantial market share in 2014 owing to numerous research in the field of bioinformatics and transcriptomics to incorporate the gene level study of the disease. The major factors attributing to the high share of the region include genomics-based biopharmaceutical research, drug design, increasing awareness for the diagnosis, and treatment in the adolescent section of the population. The presence of various economic leverages by the U.S. FDA are also expected to boost the market growth. The leverages include exemption from prescription drug fees for orphan drugs, tax credits for qualified clinical testing, and market exclusivity for seven years for diagnostics and drug manufacturing companies that are into orphan drug manufacturing. Moreover, presences of many institutional bodies that are involved in orphan disease research are expected to boost the market growth positively. These organizations are employing the novel techniques such as gene targeting to sort out the frequent gene mutations and prepare the new drug moieties for better treatment of the disorder. Asia Pacific region is expected to exhibit substantial growth over the forecast period on account of rapidly improving healthcare infrastructure in this region.
Key companies in neurometabolic disorders market include Amicus Therapeutics, ISU Abxis, JCR Pharmaceuticals, Biosidus., Greenovation Biotech, UAB Proforma, Dong-A Socio Group, ExSAR Corporation, Lixte Biotechnology Holdings, Inc, Neuraltus Pharmaceuticals, Protalix, Pharming Group N.V., Protalix BioTherapeutics, Amicus, Biomarin, Genzyme, Shire, Greencross. Launch of novel drugs is the major strategy adopted by the market players to capture significant share. The key market players are adopting competitive marketing strategies to maintain their market share such as the introduction of combination therapies like fabrazyme and replagal with less immunogenicity reactions by Amicus Therapeutics. Special incentives from the government in the form of guiding, funding, and market exclusivity make it a convincing opportunity for vendors to conduct research and develop versatile therapies.