[GUEST ACCESS MODE: Data is scrambled or limited to provide examples. Make requests using your API key to unlock full data. Check https://lunarcrush.ai/auth for authentication information.]  Persimmon Tree Investments [@PersimmonTI](/creator/twitter/PersimmonTI) on x 9566 followers Created: 2025-07-06 02:22:52 UTC $XBI — Orphaned No More $RARE / $MREO, $SRRK, $BBIO, $ALNY, $RCKT, $BMRN Below, the final language of the Orphan exclusivity expansion language that passed as part of the final megabill. Note that in addition to Medicare price negotiation exemption being extended to Tx that treat multiple rare disease indications, rare disease Tx would have a delayed start to the price negotiation countdown if the reason it lost exemption was that a non-rare-disease indication had been added to its label. Playing this out with a specific hypothetical, $RARE / $MREO Setrusumab, we can see how these provisions could have significant beneficial impact on orphan Tx exclusivity: - Setrusumab (hypothetically) receives approval for Osteogenesis Imperfecta, starting its period of orphan exclusivity. - $RARE pursues Setrusumab approval in additional rare bone disease indication, such as Juvenile Paget’s Disease. Should it receive orphan designation and approval for the indication, this sequential orphan rare disease approval would stack on the original orphan exclusivity. And so on for any other rare bone disease indication. - Then, after all rare disease indications for Setrusumab had been exhausted, perhaps $RARE could pursue Setrusumab for the non-rare indication of osteoporosis, which if approved, would start the standard XX year exclusivity period for a biologic. Not necessarily a realistic scenario but perhaps illustrative of the added flexibility, and potential life cycle value, added to orphan drugs by this measure. And we could run the same hypothetical for $SRRK and its apitegromab: - initial rare disease indication, SMA - stack on orphan exclusivity for other rare muscle disease, such as muscular dystrophy. - once all rare muscle disease indications have been prosecuted, perhaps explore anti-myostatin in non-rare indication of lean muscle preservation during weight loss. .  XXXXXX engagements  **Related Topics** [drugs](/topic/drugs) [budgeting](/topic/budgeting) [$rckt](/topic/$rckt) [$alny](/topic/$alny) [$mreo](/topic/$mreo) [$rare](/topic/$rare) [$xbi](/topic/$xbi) [$psnl](/topic/$psnl) [Post Link](https://x.com/PersimmonTI/status/1941684242066358445)
[GUEST ACCESS MODE: Data is scrambled or limited to provide examples. Make requests using your API key to unlock full data. Check https://lunarcrush.ai/auth for authentication information.]
Persimmon Tree Investments @PersimmonTI on x 9566 followers
Created: 2025-07-06 02:22:52 UTC
$XBI — Orphaned No More
$RARE / $MREO, $SRRK, $BBIO, $ALNY, $RCKT, $BMRN
Below, the final language of the Orphan exclusivity expansion language that passed as part of the final megabill.
Note that in addition to Medicare price negotiation exemption being extended to Tx that treat multiple rare disease indications, rare disease Tx would have a delayed start to the price negotiation countdown if the reason it lost exemption was that a non-rare-disease indication had been added to its label.
Playing this out with a specific hypothetical, $RARE / $MREO Setrusumab, we can see how these provisions could have significant beneficial impact on orphan Tx exclusivity:
Setrusumab (hypothetically) receives approval for Osteogenesis Imperfecta, starting its period of orphan exclusivity.
$RARE pursues Setrusumab approval in additional rare bone disease indication, such as Juvenile Paget’s Disease. Should it receive orphan designation and approval for the indication, this sequential orphan rare disease approval would stack on the original orphan exclusivity. And so on for any other rare bone disease indication.
Then, after all rare disease indications for Setrusumab had been exhausted, perhaps $RARE could pursue Setrusumab for the non-rare indication of osteoporosis, which if approved, would start the standard XX year exclusivity period for a biologic.
Not necessarily a realistic scenario but perhaps illustrative of the added flexibility, and potential life cycle value, added to orphan drugs by this measure.
And we could run the same hypothetical for $SRRK and its apitegromab:
- initial rare disease indication, SMA
- stack on orphan exclusivity for other rare muscle disease, such as muscular dystrophy.
- once all rare muscle disease indications have been prosecuted, perhaps explore anti-myostatin in non-rare indication of lean muscle preservation during weight loss.
.
XXXXXX engagements
Related Topics drugs budgeting $rckt $alny $mreo $rare $xbi $psnl