Morquio Syndrome (MPS-IV) Drug Industry: Global Progress on Developing a Drug for Morquio Syndrome

Morquio Syndrome (MPS-IV) Drug

Morquio syndrome (MPS-IV) drug industry, also known as Mucopolysaccharidosis type IV (MPS-IV), is a rare lysosomal storage disease caused by genetic mutations that prevent the body from properly breaking down molecules called glycosaminoglycans (GAGs). When GAGs accumulate in cells, it causes damage that impacts the entire body and leads to serious health issues. For many years, Morquio syndrome had no approved drug treatment options and those suffering lived with significant health challenges and reduced life expectancy. However, in recent times there have been major advancements in developing a potential disease-modifying drug that could help Morquio syndrome patients worldwide.

Enzyme Replacement Therapy Development

Scientists have been working to develop an enzyme replacement therapy (ERT) for Morquio syndrome after discovering the underlying genetic cause - a deficiency in the enzyme N-acetylgalactosamine 6-sulfatase (arylsulfatase B, or ARSB). ERT aims to supplement the missing or malfunctioning enzyme through intravenous infusions to help break down GAG storage in cells.

In 2015, Sanfilippo syndrome drug Naglazyme was approved for use in Morquio Syndrome (MPS-IV) Drug after clinical trials found it helped reduce urine GAG levels when administered biweekly. However, it only slowed progression rather than fully addressing the underlying problems. Researchers continued their efforts to engineer a tailored ERT specifically for Morquio syndrome.

Velmanase alfa is a recombinant human ARSB enzyme developed by Ultragenyx Pharmaceutical through extensive research and clinical testing. It completed Phase 1/2 and Phase 3 global clinical trials involving over 100 Morquio syndrome patients and proved safe and effective in reducing urine GAG levels by 70-80% on average. The drug's Biologics License Application was accepted for review by the U.S. Food and Drug Administration in late 2018.

Morquio Syndrome (MPS-IV) Drug Industry and Global Access

In 2020, velmanase alfa received marketing authorization from the European Commission under the trade name Mepsevii, marking the first approved treatment for Morquio syndrome in Europe. This paved the way for national reimbursement decisions and access to the drug across European countries.

In 2021, Mepsevii was approved for use in Morquio syndrome by the FDA in the United States as well. This granted many long-waiting American patients the opportunity to receive ERT treatment through their insurance plans or alternative funding options.

Ultragenyx is working closely with regulatory bodies worldwide to expand velmanase alfa's availability to more countries and regions affected by Morquio syndrome. Marketing applications have been submitted to health authorities in several nations including Brazil, Canada, Japan, Taiwan, and the United Arab Emirates. Approvals are pending or anticipated in the near future that will help global Morquio syndrome patients gain regulated access to the first disease-modifying therapy.

Improving Quality of Life Outcomes

The approval and early usage of velmanase alfa ERT has provided encouraging results for managing Morquio syndrome complications and improving patient prognosis. Early reports from Europe and the United States found therapy led to stabilized walking ability and lung function, fewer orthopedic surgeries, and relief from painful skeletal dysplasia issues over the first few years of treatment.

Patients have reported better endurance, mobility, breathing and reduced day-to-day discomfort—drastically enhancing their quality of life. While Morquio syndrome remains currently incurable, velmanase alfa offers new hope by slowing or halting disease progression when treatment begins early in life. Long-term follow-up studies will continue to evaluate therapy benefits on survival rates and outcomes into adulthood for this young patient population.

Looking to the Future

With the first approved drug therapy now accessible to large segments of the global Morquio syndrome community, future research can build upon this important milestone. Ultragenyx is working to expand ERT indications to a broader age range including very young pediatric patients. Gene therapy is also being explored as a potential one-time curative approach versus lifelong ERT.

Regulatory approvals spread velmanase alfa availability worldwide, more clinics and centers will gain expertise in administering and monitoring this specialized treatment. This will further optimize patient management. The biopharma industry's achievement in developing the first disease-modifying drug for Morquio syndrome has brought the promise of a brighter future closer for families touched by this rare and debilitating disease.

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