Ornithine Transcarbamylase (OTC) Deficiency Treatment Market Size (2024 - 2029)

Ornithine Transcarbamylase Deficiency Treatment Market Analysis

The Ornithine Transcarbamylase Deficiency Treatment Market size is estimated at USD 0.82 billion in 2024, and is expected to reach USD 1.01 billion by 2029, growing at a CAGR of 4.30% during the forecast period (2024-2029).

Ornithine transcarbamylase (OTC) deficiency is a rare X-linked genetic disorder showing a complete or partial lack of the ornithine transcarbamylase (OTC) enzyme. The rising approvals for novel pipeline products for ornithine transcarbamylase (OTC) deficiency treatment are expected to influence market growth. In March 2024, iECURE received the Medicines & Healthcare Products Regulatory Agency (MHRA) approval for its clinical trial authorization application (CTA) to expand the phase I/II OTC-HOPE clinical trial of its investigational gene editing-based therapy, ECUR-506, in the United Kingdom.

In addition, in August 2023, Bloomsbury Genetic Therapies Limited received orphan drug designation from the US Food and Drug Administration (FDA) for the company's investigational liver-targeted gene therapy, BGT-OTCD, for the treatment of ornithine transcarbamylase deficiency.

Furthermore, growing awareness for ornithine transcarbamylase deficiency disease and increasing campaigns organized by public and private entities are expected to fuel market growth. For instance, the NIH's National Center for Advancing Translational Sciences (NCATS) and the NIH Clinical Center sponsor the event, Rare Disease Day, on the last day of February, i.e., 28 or 29, as a part of the global Rare Disease Day observance every year. This day celebrates and includes raising awareness about rare diseases, the people they affect, etc. NIH conducted this event on February 29, 2024, and held it in person at the NIH Main Campus (Natcher Conference Center), Bethesda, United States.

In addition, favorable reimbursement scenarios in developed countries and patient assistance programs, particularly in developing regions, have also been fueling the growth of the OTC deficiency treatment market in recent years. For instance, in January 2024, Medunik USA, a member of Duchesnay Pharmaceutical Group (DPG), announced that Pheburane coverage is added to the Medicaid Preferred Drug List in 10 states with the recent addition of Indiana, Tennessee, Georgia, and Missouri, along with those already in effect in New Hampshire, New Mexico, North Dakota, Oklahoma, Texas, and Utah. Pheburane is also included on the California Contract Drugs List, and no prior authorization is required for the drug. Hence, it is easily accessible to patients with reimbursement. The drug is covered by commercial insurance plans, mainly offered by private players, and is covered by employers, which constitutes around 80% of the total commercially insured US population.

Medunik USA offers a patient program called the UNIK Support Program for Pheburanr, which provides specialized services, including a copay savings program, patient care liaison services, mail-order pharmacy, and other support services. Thus, the factors above are expected to boost the market growth.

However, the high cost of drugs, limited budget, and less awareness of disease and treatment in lower-income countries may restrain the market growth.

Ornithine Transcarbamylase Deficiency Treatment Industry Segmentation

Ornithine transcarbamylase deficiency is a rare genetic condition that results in ammonia building up in the blood. This condition is more prominently seen in boys than girls and tends to be more severe when symptoms emerge shortly after birth. Ornithine transcarbamylase deficiency is caused by changes in the OTC gene, which instructs the body to make the OTC enzyme. In OTC deficiency, the OTC gene is either damaged or missing.

The ornithine transcarbamylase (OTC) deficiency treatment market is segmented into treatment type, route of administration, distribution channel, and geography. The market is segmented by treatment type into glycerol phenylbutyrate, sodium phenylbutyrate, sodium phenylacetate and sodium benzoate, and other treatment types. By route of administration, the market is segmented into oral and intravenous. By distribution channel, the market is segmented into hospital pharmacies, retail pharmacies, online pharmacies, and other distribution channels. By geography, the market is segmented into North America, Europe, Asia-Pacific, and Rest of the World. The report also offers the market sizes and forecasts for 13 countries across the region. For each segment, the market size and forecast are provided in terms of value (USD).