Resulting from a complete absence of α-Gal, Fabry disease can also occur in a more severe form called as classic Fabry. The market presents a huge opportunity in its treatment and cure.
The Fabry Disease Market is going to witness rapid growth owing to an increase in spending and regulatory assistance. Special incentives in the form of funding, guidance, and market exclusivity, make it an attractive opportunity, for vendors to conduct research.
According to TechNavio, the Global Fabry Disease Market is growing at a rapid rate and is expected to post a CAGR of 18.08 percent from 2015-2019. The Global Fabry Disease Market accounted for a share of almost one quarter of the Global Lysosomal Storage Diseases market in 2014, and it is expected to increase.
Currently, there is a lack of disease-modifying therapy in the market. It is a market which is currently dominated by Fabrazyme, and would witness further improvement, in the future.
TechNavio analysts have identified three key drivers that are aiding growth in The Global Fabry Disease Market.
Special Provisions for Orphan Drugs
Diseases with limited patient population, such as Fabry disease are usually supported by few approved drugs. Fewer patients means a smaller market for drugs, which might not be profitable enough to recover the R&D expenses of the drug.
To compensate for this limitation, regions such as the US and the EU have a provision for orphan drug designation. In the EU, orphan drugs enjoy protocol assistance and a 10-year market exclusivity. In the US, orphan designation for a drug replicates into incentives such as tax credits for qualified clinical testing, exemption from prescription drug fees unless indicated for additional non-orphan indications, and market exclusivity for seven years.
Market exclusivity is a commercially beneficial provision, which is increasingly attracting pharmaceutical companies to work on drugs for orphan diseases. In addition, the entry of generic drugs is barred for the duration of market exclusivity, thus protecting the market sales of an orphan drug.
Designated ICD-10 Code
ICD is a standard tool used for diagnostic and analytical purposes. These codes can be used to emphasize the need for patient visits to medical centers. These codes can also be used by insurance companies to understand the frequency of medical visits recommended for patients.
Therefore, these codes can be used for reimbursement purposes by private agencies or government insurers. The code for Fabry disease is E75.21 ICD-10-CM code. ICD-10-CM codes offer various advantages over ICD-9 codes. ICD-10 codes have been designed to provide enhanced specificity and better clinical details than ICD-9 codes.
These codes ensure better interpretation of physician’s documentation by auditors, third parties, and attorneys. ICD-10-CM codes are also anticipated to provide comprehensive healthcare data, thus promoting the development of advanced disease protocols and clinical pathways.
Potential Advantages of Migalastat
Migalastat (Amicus Therapeutics) is undergoing development for the treatment of patients with Fabry disease, as a monotherapy and combination therapy. Migalastat is a molecular chaperone and is anticipated to have better bioavailability than the currently approved Fabry disease ERTs: Fabrazyme and Replagal.
The latter drugs are proteinaceous in nature and, therefore, have limited penetration in some cells and tissues. On the other hand, migalastat is a small molecule, which has demonstrated high bioavailability, and is expected to penetrate all the clinically relevant target cells in Fabry patients. Migalastat is also distributed adequately inside the body. Fabrazyme and Replagal require intravenous administration leading to inconvenience for Fabry patients.
However, migalastat might overcome this inconvenience because of its oral administration. Migalastat is expected to be free from the immunogenicity reactions associated with Fabrazyme and Replagal. Co-administration of migalastat and approved ERTs might enhance the transportation of these enzymes into lysosomes, thereby increasing their efficacy.
