Transthyretin Stabilizers Market Size, Share, and COVID-19 Impact Analysis, By Drug Type (Tafamidis, Inotersen, and Others), By Route of Administration (Oral and Parenteral), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies & Drugs Stores, and Online Pharmacies), and Regional Forecast till 2032

Transthyretin is a protein made up of 127 amino acids, also known as transport protein, as it carries both thyroxine (T4) and retinol-binding protein that is bound to retinol. It is most commonly found in serum and CSF and circulates as a hometramer. Inherited mutations and aging can cause destabilization of Transthyretin (TTR). This causes the hometramer complex to break apart and form amyloid fibrils, which get deposited in organs and tissues.

Transthyretin stabilizers prevent the destabilization of transthyretin (TTR) as well as dissociation into amyloid fibrils, which avoids the further progression of TTR amyloidosis. The inherited mutations and aging leading to TTR destabilization fuels the product adoption, propelling the market growth.

Furthermore, transthyretin (TTR) stabilizers are generally well tolerated, with UTIs, diarrhea, vaginal infections, and upper abdominal pain reported as the most common side effects. Moreover, stabilization of the transthyretin (TTR) tetramer has become an essential target for novel therapies to slow down the ATTR amyloidosis progression.

• For instance, according to data published by MJH Life Sciences, in March 2021, the estimated prevalence of hATTR amyloidosis was approximately 50,000 across the globe.

Moreover, prominent players in the market are increasing R&D activities and developing new products for the treatment of hATTR-PN. Furthermore, they are involved in strategic partnerships and collaborations to increase their market presence, thereby driving the growth of the global market.

• For instance, in August 2023, BridgeBio’s Phase 3 study, ATTRibute-CM, showed positive results for acoramidis in treating ATTR cardiomyopathy (ATTR-CM). This offers hope for patients to both preserve the cardio-protective benefits of TTR and tackle the root cause of disease.

Impact of COVID-19 on the Transthyretin Stabilizers Market

The pandemic led to delays in the diagnosis of transthyretin amyloidosis (ATTR), which is the condition that transthyretin (TTR) stabilizers are used to treat. These delays were due to the patients with ATTR may have symptoms that are similar to those of COVID-19, such as fatigue, shortness of breath, and cough. Additionally, many hospitals and clinics have reduced their services during the pandemic, making it more difficult for patients to get appointments. The pandemic has also disrupted the supply chains for transthyretin (TTR) stabilizers. The disruption was recorded as the manufacturing of these drugs often relies on raw materials and components from other countries. Additionally, the closure of borders and restrictions on travel have made it more difficult to transport transthyretin (TTR) stabilizers to patients.

• According to the National Center for Biotechnology Information (NCBI), diagnostic testing for amyloidosis was restricted at several centers due to the hospital & clinic closures and a frequent shift in resources toward COVID-19 management. (May 2021)


• According to a study published by the NCBI, in August 2022, 33 ATTR patients were diagnosed in 2019, 38 in 2020, and 27 in 2021.

Segmentation

Key Insights

The report covers the following key insights:

• Key Industry Developments (Mergers, Acquisitions, Partnerships, and Others)


• Pipeline Analysis


• Impact of COVID-19 on the Market

Analysis by Drug Type

Based on drug type, the tafamidis segment held the largest revenue share of the global transthyretin stabilizers market. Tafamidis is a transthyretin (TTR) stabilizer that is approved for the treatment of transthyretin amyloidosis polyneuropathy (hATTR-PN) and transthyretin amyloidosis cardiomyopathy (hATTR-CM). It is the only drug approved for both indications. Tafamidis has been shown to be very effective in reducing the progression of both hATTR-PN and hATTR-CM.

• In the ATTR-ACT phase 3 multicenter international randomized controlled trial, it was found that 264 patients treated with tafamidis had lower rates of all-cause mortality and cardiovascular hospitalizations compared to 117 patients receiving placebo over 30 months.

The inotersen segment is projected to expand at a noteworthy CAGR during the forecast period. It is a Transthyretin (TTR)-directed antisense nucleotide that was approved by the FDA in 2018 for the treatment of polyneuropathy. It has shown an improvement in neurologic disease and quality of life in individuals with hereditary transthyretin amyloidosis. Furthermore, an increase in the prevalence of hATTR-PN is expected to boost the segment growth.

• In January 2020, according to an article published by ‘The Journal of Neurology’, the prevalence of ATTRv with polyneuropathy is approximately 10,000 (1/1,000,000 individuals).

Regional Analysis

North America is expected to hold the largest share in the transthyretin stabilizers market and maintain its dominant position during the forecast period. The growth is mainly influenced by the high prevalence of ATTR amyloidosis in the region and the availability of treatment options.

• For instance, according to data published by MJH Life Sciences, in March 2021, the estimated prevalence of hATTR amyloidosis was approximately between 10,000 and 15,000 cases in the U.S.

The Asia Pacific market is projected to grow at the fastest rate over the forecast period. This regional growth is primarily due to the increasing awareness of ATTR amyloidosis in the region and the growing demand for treatment options.

Key Players Covered

The report includes the profiles of key players such as Pfizer Inc., Ionis Pharmaceuticals, Alnylam Pharmaceuticals, Inc., and others.

Key Industry Developments

• In March 2023, Ionis Pharmaceuticals received FDA approval for eplontersen’s new drug application for treating hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).


• In September 2022, Alnylam Pharmaceuticals, Inc. reported positive results from the APOLLO-B Phase 3 Study of Patisiran in patients with ATTR-CM.


• In May 2019, Pfizer Inc. announced that their VYNDAQEL (tafamidis meglumine) and VYNDAMAX (tafamidis) received U.S. FDA approval for the treatment of cardiomyopathy of both hereditary type and wild-transthyretin-mediated amyloidosis (ATTR-CM) in adults.