Tyrosinemia Type 1 Treatment Market Size, Share, and Industry Analysis, By Drug (Nitisinone and Others), By Dosage Form (Tablet, Capsule, and Suspension), By Distribution Channel (Hospital Pharmacies, Specialty Pharmacies, and Others), and Regional Forecast, 2025-2032

Tyrosinemia type I (HT-1) is a rare genetic metabolic disorder that is marked by the deficiency of the enzyme fumarylacetoacetate hydrolase (FAH), which is necessary for the breakdown of the amino acid tyrosine. Tyrosinemia type I is typically present in infants in either acute or chronic form. Infants with an acute form of tyrosinemia experience symptoms within the first months of birth. It is more common and severe than the chronic form. The chronic form of tyrosinemia has a slower onset and less severe symptoms. It typically occurs after six months of birth. The symptoms of tyrosinemia type I often differ from person to person.

The treatment for tyrosinemia type 1 typically involves a multifaceted approach, which includes medication, dietary restrictions, and, in severe cases, liver transplantation. The market growth is driven by increasing regulatory approvals for drugs used in the treatment of Tyrosinemia Type 1 disorder.

• For instance, in June 2022, Analog Pharma and Dipharma S.A. announced that the abbreviated new drug application for Nitisinone received approval from the U.S. FDA. This generic drug is administered to adult and pediatric patients for the treatment of hereditary tyrosinemia type 1.

In addition, the development and launches of innovative medicines for the treatment of Tyrosinemia Type 1 is being pursued by a number of key market players. This is one of the factors contributing to the market growth across the world.

• For example, in November 2019, WEP Clinical, the specialist pharmaceutical services company, announced that it had launched Nitisinone MDK in the U.K., France, Germany, Italy & Poland.

Furthermore, increased awareness among both healthcare professionals and patients regarding rare diseases, such as tyrosinemia type 1, has resulted in enhanced rates of diagnosis, consequently fueling the demand for treatment options for the condition.

The COVID-19 pandemic had a negative impact on the global tyrosinemia type 1 treatment market in 2020. The COVID-19 pandemic caused disruptions in healthcare services that led to the cancellations of appointments and caused further challenges in accessing essential treatments. Some of the patients with rare inherited metabolic disorders were highly susceptible to the development of COVID-19 infection. In spite of these challenges, some of the key players, such as Cycle Pharmaceuticals Limited, introduced the Wellbeing Program to support patients with hereditary tyrosinemia type 1 disorder undergoing treatment with NITYR (Nitisinone) tablets during the COVID-19 pandemic.

Segmentation

Key Insights

The report covers the following key insights:

• Prevalence of Tyrosinemia Type 1, By Key Regions/Countries, 2022/2023
• New Product Launches, By Key Players
• Key Industry Developments - Mergers, Acquisitions & Partnerships
• Impact of COVID-19 on the Global Tyrosinemia Type 1 Treatment Market

Analysis by Dosage form

Based on the dosage form, the market is segmented into tablet, capsule, and suspension.

The capsule segment is anticipated to mark significant growth in the market over the study period. The growth of the segment is attributed to several advantages associated with capsule dosage forms, such as the ease of swallowing and masking of unpleasant taste and the odor of the medication. Due to these factors, the patients prefer capsules over tablets and suspensions. Also, increasing regulatory approvals and product launches, which are in the capsule dosage form, are supporting the segment's growth during the forecast period.

• For instance, in February 2024, Eton Pharmaceuticals announced the launch of Nitisinone capsules for the treatment of Tyrosinemia Type 1. This drug is available in specialized pharmacies and used in combination with a restricted tyrosine and phenylalanine diet.
• Similarly, in September 2019, the U.S. FDA approved the first generic version of Nitisinone capsules (Orfadin, Sobi) to treat patients with hereditary Tyrosinemia Type 1 in combination with a dietary restriction on tyrosine and phenylalanine.

Furthermore, the tablet segment is anticipated to grow at a notable growth rate in the coming years. The segmental growth is primarily driven by some companies undergoing strategic initiatives to provide medicines in developing countries through several healthcare programs.

• For instance, in January 2024, Cycle Pharmaceuticals Ltd and Inceptua Group entered into a collaboration to make NITYR (Nitisinone) tablets available through a Free Goods Programme for eligible patients with Hereditary Tyrosinemia Type 1. Through this program, medicines are being provided to different developing countries, such as India, Bangladesh, Pakistan, and others.

Furthermore, the suspension segment held a considerable market share in 2023 due to increasing product launches and patent filing.

• For example, in January 2016, the Swedish Orphan Biovitrum AB announced that the European Patent Office granted a European patent for the Orfadin (Nitisinone) oral suspension formulation, which was approved by the European Commission for the treatment of Tyrosinemia type-1 in 2015.

Regional Analysis

To gain extensive insights into the market, Download for Customization

Based on region, the Tyrosinemia Type 1 Treatment market is divided into North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa. North America accounted for a substantial market share in 2023 and is anticipated to maintain its position over the forecast period. The region's dominant market position is owing to the growing prevalence of Tyrosinemia Type 1 disorder, ultimately increasing the demand for medications in the North American region.

• For example, as per the data provided by the National Organization for Rare Disorders, Inc. in September 2019, the global prevalence of HT-1 was 1 in 100,000, but in Canada, it was 1 in 16,000 for the year 2019.

Furthermore, some key market players are focusing on receiving regulatory approvals to introduce effective medicines for the treatment of Tyrosinemia Type 1 disorder. This is one of the factors that has contributed to regional growth in the Tyrosinemia Type 1 Treatment market.

• For instance, in October 2023, Eton Pharmaceuticals announced the acquisition of the FDA-approved ultra-rare disease product, Nitisinone, which was developed by Oakrum Pharma, LLC. This acquisition expanded Eton's Tyrosinemia Type 1 treatment market product portfolio.
• Similarly, in April 2018, Sobi, a biopharmaceutical company, received approval from Health Canada for once-daily dosing of Orfadin (Nitisinone) for the treatment of HT-1.

Key Players Covered

The report includes the profiles of key players, such as Swedish Orphan Biovitrum AB, CYCLE Pharmaceuticals Limited, Analog Pharma, Dipharma S.A., Eton Pharmaceuticals, and others.

Key Industry Developments

• In June 2023, Analog Pharma and Dipharma S.A. announced the U.S. FDA approval for Nitisinone capsules 20 mg, a generic version of Swedish Orphan Biovitrum's Orfadin.
• In July 2022, Doppel Pharma CDMO delivered the first commercial batches of generic Nitisinone capsules Dipharma S.A. This medicine is used to treat adult and pediatric patients with a confirmed diagnosis of HT-1.
• In October 2020, Cycle Pharmaceuticals Limited announced that it had received approval from the Israeli regulatory authority for NITYR (Nitisinone) tablets.
• In September 2020, Swedish Orphan Biovitrum AB received a positive opinion from the Committee for Medicinal Products for Human Use for the drug Orfadin (Nitisinone) used in treating HT-1.