In light of National Thyroid Awareness Month, GlobalData highlights that the lack of awareness around hypothyroidism (also known as ‘underactive thyroid’) is impacting diagnosis rates and limiting investment. The leading data and analytics company notes that only 14%* of Phase I-III clinical trials for hypothyroidism in the last five years were sponsored by pharma companies.

Fiona Chisholm, Pharma Analyst at GlobalData comments: “A mere 14% of investment coming from pharma is extremely low, considering that industry sponsors approximately 41% of Phase I-III trials for hormonal disorders generally. There is a great need for innovation and new therapies in this area, but without greater awareness and diagnosis, opportunity will remain low.”

During National Thyroid Awareness Month, the American Thyroid Association promotes campaigns to educate the public on the importance of thyroid health, as well as the prevention, treatment, and cure of thyroid-related diseases—of which hypothyroidism is the most common. Caused by insufficient production of the hormone thyroxine T4, treatment of hypothyroidism usually involves hormone replacement therapy with oral levothyroxine, a synthetic version of T4. Dosing is individualized, based on factors such as age, sex, body mass, and residual thyroid function. However, treatment can often be challenging.

Chisholm explains: “Levothyroxine has a narrow ‘therapeutic index’—a narrow window where doses are effective and dosing outside this window produces side effects. A careful dose titration is necessary, where the dose is started low and slowly raised to find the right level. This may also require constant adjustment in response to physiological changes such as aging and weight change.

“Due to the limitations of levothyroxine, both over- and under-dosing is common, which places patients at risk of adverse consequences of treatment. For example, over-dosing of levothyroxine can cause side effects such as heart palpitations and anxiety.”

As generic levothyroxine has long dominated the market, commercial incentives for pharma companies to enter this space are limited. This is also compounded by a limited awareness of hypothyroidism among the general public, which contributes to low rates of diagnosis and treatment.

However, GlobalData notes that there is some degree of industry investment in new therapies for hypothyroidism. A notable industry player within the research & development (R&D) landscape is XERIS Pharmaceuticals, which is developing a novel subcutaneous formulation of levothyroxine, XP-8121. This drug has the potential to mitigate some of the limitations associated with oral levothyroxine that can lead to suboptimal therapeutic effects, including absorption variation, which can present challenges for establishing an ideal dose. The company expects preliminary results for its Phase I trial of XP-8121 in Q1 2022.

Chisholm concludes: “Increased rates of diagnosis and treatment could spur greater R&D investment in this field by enhancing the commercial incentives for companies active within this space. This underlines the importance of campaigns that promote greater public awareness of hypothyroidism and other thyroid disorders.”

*Includes Phase I, Phase I/II, Phase II, Phase II/III, Phase III and Phase III/IV trials initiated between January 2017 and December 2021, including completed, ongoing, suspended, and withdrawn trials.