Propionic Acidemia Therapeutics will grow at highest pace owing to growing research funding for rare disease treatment.

 

Propionic acidemia is an inherited genetic disorder that affects how the body breaks down certain protein-containing foods. It occurs due to the deficiency of an enzyme called propionyl-CoA carboxylase (PCC) that helps break down protein. As a result, toxic levels of propionic acid and ammonia build up in the body which can damage the nervous system resulting in poor muscle tone, seizures, intellectual disabilities and cardiac arrest if left untreated. Currently, treatment options are limited and focus on managing acute episodes and preventing long term complications through special diet, vitamin B12 injections, medication to lower ammonia levels and liver transplant.

The global propionic acidemia therapeutics market is estimated to be valued at US$50 million in 2024 and is expected to exhibit a CAGR of 5% over the forecast period 2024-2032.

Key Takeaways

Key players operating in the propionic acidemia therapeutics market include Synlogic, Mirum Pharmaceuticals, Recordati Rare Diseases Inc. among others. Companies are investing heavily in developing new drug therapies for orphan diseases to address the high unmet needs.

The growing diagnosis rate of propionic acidemia owing to expanding newborn screening programs and availability of advanced genetic testing is expected to fuel the market growth. According to CARES foundation, nearly 1 in 250,000 newborns are affected by propionic acidemia globally each year.

Technological advancements in gene therapy, drugs targeting metabolic pathways and precision medicine holds potential to transform the treatment landscape of propionic acidemia in the coming years. Researchers are exploring gene therapy as a potential cure to supplement the defective PCC enzyme in patients.

Market Trends

Increasing focus on the development of gene therapies - Several biotechs are evaluating gene therapy as a potential cure for propionic acidemia with the goal of supplementing the faulty PCC gene. This represents a paradigm shift from conventional disease management to a potential one-time curative approach.

Growing research on PCC supplements - Researchers are conducting preclinical studies to evaluate oral PCC enzyme supplements as an adjunct to diet therapy. PCC supplements aim to normalize propionic acid and ammonia levels in patients to reduce complications and improve long term outcomes.

Market Opportunities

Orphan drug designation and research funding - Government agencies and nonprofit organizations are supporting research on rare diseases like propionic acidemia through grants, tax incentives and expedited review process which is attracting more companies.

Unmet needs in neonatal screening and long term management - There remains significant scope to improve newborn screening, long term monitoring and outcome data which can support the evaluation of new therapies. Establishing clinical care guidelines can also enhance standards of care.

Impact of COVID-19 on Propionic Acidemia Therapeutic Market Growth

The COVID-19 pandemic has greatly impacted the global Propionic Acidemia Therapeutic market. During the initial outbreak, there was a steep decline in the number of new patient diagnoses and hospital admissions due to postponed treatments and lockdowns across regions. This led to reduced demand for Propionic Acidemia Therapeutic drugs in 2020. However, with increasing remote consultations and reopening of healthcare facilities under strict safety guidelines post lockdowns, the market is recovering gradually.

The remote monitoring of patients and telemedicine are helping healthcare providers to continuously monitor patients' health status and provide necessary consultation during this crisis. Several pharmaceutical companies are adopting collaborative approaches and accelerating their drug development processes to find potential therapeutics for COVID-19. Researchers are also investigating the effectiveness of existing Propionic Acidemia Therapeutic drugs such as sodium phenylbutyrate in treating coronavirus infected patients. If found effective, it will provide a new treatment option and boost the market demand. The pandemic has highlighted the need for development of novel drugs and services focusing on disease management to minimize disruption of care in future pandemic situations.

Geographical Regions with High Propionic Acidemia Therapeutic Market Value

North America holds the leading share of the global Propionic Acidemia Therapeutic market in terms of value, owing to the presence of major pharmaceutical players, high patient awareness levels, and availability of advanced treatment options in the region. Within North America, the United States contributes maximum to the regional market value. Europe follows North America in terms of market size due to supportive government policies for orphan drug development and rising healthcare spending. Asia Pacific is expected to witness the fastest growth during the forecast period majorly driven by improvements in the diagnostics and healthcare infrastructure across emerging countries.

Fastest Growing Regional Market for Propionic Acidemia Therapeutics

Asia Pacific region presents lucrative opportunities for Propionic Acidemia Therapeutic drug manufacturers globally. Factors such as the rising patient population, growing healthcare expenditure, and increasing focus of international players to tap new markets will support the growth. Governments are investing heavily to strengthen the healthcare systems and offer improved treatment facilities. This presents an ideal environment for companies to launch new drugs. Countries like China and India serve as high potential markets within Asia Pacific, exhibiting high unmet medical needs and a rapidly expanding patient pool. Besides geographical expansion, companies are also exploring strategic collaborations with regional players to boost their market presence across Asia Pacific.

Get more insights on this topic : https://nouw.com/ajay100/propionic-acidemia-therapeutics-market-is-estimated-to-witness-high-growth-owing-to-increasing-rd-38619788

What Are The Key Data Covered In This Propionic Acidemia Therapeutics Market Report?

:- Market CAGR throughout the predicted period

:- Comprehensive information on the aspects that will drive the Propionic Acidemia Therapeutics's growth between 2024 and 2031.

:- Accurate calculation of the size of the Propionic Acidemia Therapeutics and its contribution to the market, with emphasis on the parent market

:- Realistic forecasts of future trends and changes in consumer behaviour

:- Propionic Acidemia Therapeutics Industry Growth in North America, APAC, Europe, South America, the Middle East, and Africa

:- A complete examination of the market's competitive landscape, as well as extensive information on vendors

:- Detailed examination of the factors that will impede the expansion of Propionic Acidemia Therapeutics vendors

About Author:

Ravina Pandya, Content Writer, has a strong foothold in the market research industry. She specializes in writing well-researched articles from different industries, including food and beverages, information and technology, healthcare, chemical and materials, etc. (https://www.linkedin.com/in/ravina-pandya-1a3984191)

*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it