Market Trends of Pompe Disease Industry
This section covers the major market trends shaping the Pompe Diseases Market according to our research experts:
Enzyme Replacement Therapy (ERT) Segment is Estimated to Witness a Considerable Growth Over the Forecast Period
Enzyme replacement therapy (ERT) is a medical treatment in which replacement enzymes are administered to patients who suffer from chronic conditions caused by enzyme deficiency or malfunction. The most common method of ERT is intravenous infusions, in which the replacement enzyme is given directly into the bloodstream through a controlled fluid drip. Replacement enzymes for ERT are derived from human, animal, and plant cells, which are then genetically modified and processed before administration to the patient. By receiving these enzyme replacements, the body can successfully perform the functions inhibited by the deficiency. This therapy is the only effective treatment wherein the enzyme alpha-glucosidase, which is deficient in Pompe disease, is given by injection. This will reduce and slow the progression of muscle wasting and heart problems.
The efficiency of enzyme replacement therapy (ERT), the increasing cases of Pompe disease, and rising research funding for rare diseases are the major drivers for the segment. For instance, according to the research study published in November 2021, the only approved treatment for Pompe disease is enzyme replacement therapy (ERT), which involves the administration of rhGAA intravenously. Therefore, the effectiveness of ERT therapy for Pompe disease, the only therapy approved by the U.S. FDA, has increased demand for enzyme replacement therapy (ERT), which is anticipated to drive segment growth. Furthermore, the product launches by the key market players with respect to the segment are also anticipated to drive market growth over the forecast period. For instance, in August 2021, the U.S. FDA approved a new enzyme replacement therapy, Nexviazyme (alglucosidase alfa-ngpt) of Sanofi Genzyme, for patients aged 1 year and older with late-onset Pompe disease.
So, the segment is expected to grow a lot over the next few years because enzyme replacement therapy (ERT) works well and new products are coming out.
North America is Expected to Witness a Significant Growth Over the Forecast Period
North America is expected to witness considerable growth over the forecast period. The growth is due to factors such as the increasing launch of novel drugs, the presence of a large number of pharmaceutical companies, growing research and development expenditure, and increasing patient preference associated with advanced therapies, product launches, and technological advancements. For instance, according to the research study published in the International Journal of Neonatal Screening in March 2021, California examined over half a million infants, with a birth frequency of 1 in every 25,200 in 2021. In addition, as per the research journal published in November 2021, the prevalence of late-onset Pompe disease (LOPD) in people across the United States, Montreal, and Canada is estimated to be 1.0%. As a result, the rise in Pompe disease prevalence is expected to boost market growth in the North American region.
Key product launches, a high concentration of market players or manufacturers' presence, acquisitions and partnerships among major players, the increasing prevalence of Pompe disease, and rising government initiatives in the United States are some of the factors driving the growth of the Pompe disease market in the country. For instance, in January 2021, The Assistance Fund (TAF) launched a new program for qualified persons living with Pompe disease to assist them and their families with out-of-pocket medical costs. The program helps with treatment-related copayments, health insurance premiums, and other medical expenses associated with the metabolic condition, which affects around one out of every 40,000 people in the United States. For instance, in February 2021, Spark Therapeutics, a member of the Roche Group, announced the dosing of the first participant in the Phase 1/2 RESOLUTESM trial of SPK-3006, an investigational liver-directed adeno-associated viral (AAV) vector gene therapy for late-onset Pompe disease (LOPD). Therefore, increasing awareness about Pompe disease and product launches are expected to fuel market growth in the country.
