Biotech: Access To TTR Medications Remains Broad, But Payer Scrutiny Is Intensifying

Research evidence excerpt

Biotech: Access To TTR Medications Remains Broad, But Payer Scrutiny Is Intensifying

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to self-administered autoinjector) adds some flexibility, but our expert thinks that CARDIO-TTRansform

showing a benefit as add-on to tafamidis is likely to be seen as a class effect (guidelines/access unlikely to

distinguish between them vs ALNY). On sHTG, our expert is excited about upcoming novel therapies from

IONS and ARWR, but access will be initially restricted to the most severe patients. On achondroplasia,

our expert notes that rare pediatric indications are usually not heavily managed given small budgetary

impact so he expects ASND to get parity access to BMRN (despite the non-insignificant premium pricing).

Amvuttra Broadly Accessible But Payer Scrutiny Intensifying - Our payer KOL described the current

TTR landscape as evolving but notes that big picture, access to Amvuttra remains solid, albeit tightly

managed. Amvuttra carries a high price tag and payers have responded accordingly with step-edits and

restrictions on combo use. On the commercial side, our KOL indicates that Amvuttra currently sits behind

a single step-edit, with Vyndamax and Attruby positioned as co-preferred on formulary. He also notes

that from a clinical standpoint it is difficult to defend a full step-edit through both stabilizers (given same

MoA) before accessing a silencer, and the current commercial framework essentially provides parity

access for BBIO and PFE (covered by RBC analyst Trung Huynh). While he found BBIO's decision to price

Attruby at a modest discount to tafamidis surprising, he views it as strategically sound overall and finds

the combination of a slightly lower price and potentially better clinical profile compelling.