Recent Phase 3 data show oral small molecules succeeding in complex indications. Roche's phenobrutinib met its endpoint in progressive multiple sclerosis, and Bridge Biopharma's Imfegratinib improved height velocity in achondroplasia. This signals a potential shift toward more convenient, patient-friendly oral therapies in areas historically reliant on injectables.
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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.
In the competitive oral SERD space for breast cancer, companies like Roche and AstraZeneca are differentiating not by proving superior degradation mechanisms but by pursuing approvals in first-line and adjuvant settings, sidestepping the crowded second-line market to find the biggest impact.
Praxis Precision Medicines is highlighted as a company with breakout potential. With promising data in both essential tremor and rare epilepsy, it could submit to the FDA and become a commercial-stage company with multiple neuroscience drugs by year-end, a rare and rapid ascent in this challenging sector.
Despite the landmark approvals of two complex gene therapies for sickle cell disease, their commercial rollout has been slow. An effective, easy-to-administer pill from Fulcrum Therapeutics could completely disrupt the market by offering a simpler, more accessible alternative, demonstrating how 'good enough' technology can beat a more complex breakthrough.
A key trend in 2025's drug approvals is that "best-in-class" therapies are distinguished not just by efficacy, but by innovations in formulation and delivery that improve the patient experience. Examples include subcutaneous versions of IV drugs and new delivery methods that expand patient access.
Protagonist believes its oral IL-23 blocker will not just compete with existing injectables but will capture a new market. They target the over 50% of eligible patients who currently take no therapy due to a dislike of injections or the safety profiles of other oral options, thereby expanding the total addressable market.
The disappointing launch of Bristol-Myers' SoTic2 created skepticism around the entire Tic2 inhibitor class. However, strong new data from Alumis and Takeda showing biologic-level efficacy is reframing the narrative, proving the mechanism is potent and creating a major new opportunity in immunology for oral therapies.
The success of Praxis's small molecule for a genetic epilepsy presents a strategic alternative to cell and gene therapies. In an era where complex modalities face funding, safety, and commercial hurdles, advanced small molecules offer a viable and potentially more practical path for treating genetic disorders.
Recent FDA approvals for Milestone's Cardamist nasal spray and J&J's subcutaneous Ribrevent Fastpro highlight a key industry trend: improving patient convenience. These products shift treatment from clinical settings to on-demand, at-home use or reduce administration time, creating value beyond just clinical efficacy.
BridgeBio aims to become a "next generational" company like Regeneron. They believe the rare combination of two ingredients makes this possible: a successful, launched flagship product generating revenue, and a robust pipeline of multiple Phase 3 programs all set to read out within a year.
