While China is introducing a 7-year orphan drug exclusivity, its impact may be limited. The critical factor for creating a viable market, mirroring the US experience, is the reimbursement environment. The ability to secure high prices for innovative therapies will ultimately determine if China becomes a primary market for orphan drugs.

Adderall's success proves a core chemical patent isn't essential for market dominance. A strong brand that becomes synonymous with a condition, combined with secondary patents on novel delivery mechanisms (like Adderall XR's capsule), can create a durable, highly profitable business moat.

The FDA incentivizes animal drug development by granting years of market exclusivity to companies that prove a generic human drug works for a novel use in animals. This avoids the "aspirin problem" in human medicine, where no one will fund trials for off-patent drugs because they can't be profitably marketed.

A key tension is emerging in orphan drug development. While the FDA's approval standards appear to be rising and becoming more erratic, Congress has simultaneously created a powerful economic incentive by exempting orphan-only drugs from Medicare price negotiation, creating a complex strategic landscape.

To overcome pharma's preference for new chemical entities (NCEs), Cereno created a second-generation drug by deuterating its original molecule. This modification improved the metabolite profile and, critically, made it a patentable NCE, opening doors for broader platform deals.

By reformulating existing oncology drugs, Nenology uses the streamlined 505(b)(2) regulatory pathway, de-risking and accelerating development. Simultaneously, their composition-of-matter patents provide strong intellectual property protection typically associated with entirely new chemical entities, creating a unique strategic advantage.

The acquisition of Verona shows that a novel mechanism of action with a substantial clinical effect can make a company a prime M&A target. This holds true even with weaknesses like no composition of matter patent or an unfashionable drug delivery method, especially in disease areas lacking innovation.

Instead of patenting a specific molecule, Alt-Pep underwent a decade-long process to patent the novel alpha-sheet protein structure itself. This unconventional IP strategy gives them a powerful, defensible platform applicable across numerous amyloid diseases, not just a single target composition.

To commercialize a simple mixture, the company built an IP portfolio around the timing, delivery, and indications for GIK. Crucially, they secured a 'trifecta' of FDA support: Special Protocol Assessment, Breakthrough Designation, and a Biologic License Application, which grants 12 years of market exclusivity, creating a strong competitive barrier without a traditional drug patent.