While a complete cure for genetically inherited, chronic diseases is yet to be discovered, continuous research is being done to provide better treatment for patients suffering from them. Two genetically inherited diseases that are a major concern for the global population are hemophilia and cystic fibrosis (CF). While hemophilia is an X-linked genetic disorder that results in excessive blood loss during injury, causing fatigue or even death, cystic fibrosis is another genetic disorder that affects a person’s health and digestive systems.
What are the chief therapies for hemophilia and cystic fibrosis?
With no cure for hemophilia, bleeding episodes can be treated through vaccinations, clot-preserving medications, fibrin sealants, and physical therapy. Gene therapy has also emerged as an effective cure for this disorder. Researchers at The American Society of Hematology (ASH) in a recent clinical trial found that after a single dose of gene therapy, patients with hemophilia achieve near-normal levels of blood clotting. Patients who underwent this clinical trial suffer from severe or moderate level of hemophilia. With blood clotting levels close to normal, patients no longer experience disabling bleeding episodes.
Afstyla, a biologic drug developed by SK Chemicals, is one of the latest drugs developed to address hemophilia. The drug treats Type A hemophilia, which is found mostly among males at birth. In this condition, the infant’s blood does not clot normally due to a missing or defective factor VII (a clotting protein). Afstyla has recently obtained regulatory approval from the US Food and Drug Administration along with Health Canada.
In the case of CF, airway clearance techniques, oxygen therapy, salt solutions, and steroids are administered for treatment. Gene therapy has emerged as an effective treatment option for CF as well. Messenger ribonucleic acid (mRNA) therapeutics is another widely used treatment for his genetic disorder. The lungs, pancreas, and the digestive system are all affected by CF. In a small, randomized clinical trial, researchers at the Karolinska Institute in Sweden found that vitamin D supplements may improve lung function in CF patients. The trial found that vitamin D supplements have a positive effect on the immune system, which might eventually lead to better respiratory function.
Why have the Americas emerged as a major market for hemophilia and CF drugs?
North and South America together hold more than half of the market share for hemophilia and CF drugs. Along with having the largest patient base, this region also has better health policies, reimbursement schemes, and patient-friendly acts in place. These include the Genetic Non-Discrimination Act (GINA), Patient Protection and Affordable Care Act (PPACA), and patients access to treatment act. Such acts and policies provide a congenial environment for pharma companies and R&D institutions to invest in CF and hemophilia drugs.
Some of the key vendors of hemophilia and CF drugs in the Americas include Baxalta, Vertex Pharmaceuticals, AbbVie, Pfizer, and Bayer.
Looking for more information on the hemophilia drugs market?
Order the 2016-2020 Report on the Global Hemophilia Drugs Market
You might also be interested in:
The 2016-2020 Report on the Global Cystic Fibrosis Market
The 2016-2020 Report on the Global RFID Blood Monitoring Systems Market
