GlobalData : BridgeBio’s acoramidis positioned to directly compete against Pfizer’s Vyndaqel for treatment of ATTR-CM if it passes trials, says GlobalData

12 Apr 2022
BridgeBio's acoramidis positioned to directly compete against Pfizer's Vyndaqel for treatment of ATTR-CM if it passes trials, says GlobalData Posted in Pharma

BridgeBio's acoramidis, an orally administered small molecule transthyretin, is currently being assessed in Phase II and Phase III trials and if it passes, it will be fighting against Pfizer's Vyndaqel (tafamidis) for a share of the transthyretin amyloidosis cardiomyopathy (ATTR-CM) and ATTR-polyneuropathy (ATTR-PN) market, observes GlobalData, the leading data and analytics company.

Samisha Khangaonkar, Pharma Analyst at GlobalData, comments: "As many early symptoms of amyloidosis can be difficult to differentiate from other diseases, diagnosing patients at an early stage is an obstacle faced by many clinicians. According to key opinion leaders interviewed by GlobalData, ATTR-CM patients were seeing an average diagnosis timeframe of 8.6 years, at which point almost 50% would already be under cardiac care. This previous symptomatic care can also be an issue as many cardiology drugs can cause an exacerbation of ATTR symptoms and lead to a clinical worsening of the associated cardiomyopathy."

Vyndaqel, which was approved in 2018, is the only certified therapy to treat ATTR-CM and ATTR-PN. Prior to its approval, there was no targeted treatment for ATTR-CM and, as a result, physicians had no incentive to push for an ATTR-CM diagnosis. Since the drug's approval, the diagnosis rate of ATTR-CM has increased.

Khangaonkar continues: "Despite Vyndaqel's efficacy and availability, its high price point is a major barrier to treatment access and it has a significant co-pay, making Vyndaqel unaffordable for many patients. Due to the age demographics of ATTR-CM patients, many physicians are opting out of Vyndaqel treatment to avoid placing the stress of payment on their patients."

In addition to its efforts in ATTR cardiomyopathy, acoramidis is set to be the only oral drug approved in the US for ATTR polyneuropathy, as Vyndaqel failed to gain approval by the FDA for this indication as they required further efficacy studies before granting approval.

As acoramidis will likely see approvals in both polyneuropathy and cardiomyopathy, this provides a greater opportunity for pricing contracts to be negotiated with payers as the drug will be able to treat a larger patient population than Vyndaqel. In addition, according to key opinion leaders interviewed by GlobalData, acoramidis, while beneficial in a similar way to Vyndaqel, looks like it may be more potent than Vyndaqel. However, due to Vyndaqel's dominance in the space, acoramidis would likely benefit from a head-to-head study.

Khangaonkar concluded: "A strong move from BridgeBio and its partner companies would be to see the company engaging in disease awareness and patient services, as seen by its main competitors. The pipeline for ATTR is growing quickly and although this brings new hope to the disease area, real-world data and long-term efficacy data are still in progress. Companies will need to differentiate their therapies from Vyndaqel, and in turn, the launch of additional therapies will greatly impact Vyndaqel's patient share and pricing strategy."