GSK opted out of WAVE's AATD program, citing a small market. However, with ~200,000 patients, this is a large rare disease. The decision, made before data, likely reflects a portfolio reprioritization under a new CEO, not a fundamental issue with the asset's commercial potential.

The pharmaceutical industry's focus on rare diseases has intensified, with 57% of all novel drugs approved in 2025 designated as orphan treatments. This is a continued increase from prior years, indicating a strategic shift towards smaller patient populations with high unmet needs, as exemplified by three different drugs for Hereditary Angioedema (HAE) being approved within ten weeks.

Dealing with regulatory bodies can be terrifying, especially for a startup facing a recall. The key is to present objective facts, demonstrate a rigorous process, and make decisions that protect the product and patient. This builds trust and ensures long-term viability.

The FDA receives raw and cleaned datasets from sponsors, not just summary reports. Their internal teams conduct independent analyses, which can lead to findings or data presentations in the official drug label that differ from or expand upon what's in the published paper.

Voyager CEO Al Sandrock supports the FDA's use of accelerated approval for severe diseases but argues it must be coupled with industry accountability. He praises Amelix for voluntarily pulling its ALS drug after a failed confirmatory trial, framing such responsible actions as essential for maintaining the FDA's willingness to be flexible with approvals based on surrogate endpoints.

Data from Pfizer's MetSera asset showed a side effect profile similar to Amgen's Meritide. This suggests tolerability issues are a temporary, upfront "price" for high-dose efficacy, reaffirming the viability of both long-acting programs and boosting confidence in the drug class.

Including patient advocates in decision-making is critical but can create strategic conflicts. A patient group advocated for unblinding a trial early for faster access, a move that pleased the market but was criticized by regulators for potentially compromising long-term survival data.

When asked if they would investigate a safety concern for a specific drug at an external party's request, the FDA expressed reluctance. Such an analysis would raise questions of bias. Instead, they prefer to address these questions by pooling data from multiple drugs with a similar mechanism of action.

Patient advocates for a Huntington's therapy are frustrated not just by the FDA's halt, but by its reversal on previously agreed-upon trial design. The agency initially accepted an external control arm but later deemed it inadequate, creating regulatory uncertainty that erodes trust and could chill future development in rare diseases.