AZ Latina ☮️🌊🟦

The FDA has the chance to put its new Rare Disease Framework into action by granting the requests in the 313-pg Citizens' Petition and 330+ pg legal memo filed on Docket # FDA-2025-P-2109, much of which is based on real world evidence. Like many #raredisease patients, people in the #ALS community had to become citizen scientists. We collected ~ 8 yrs RWE after a 28-week P3 RCT ended in 2020. Some data came from additional dosing in EAP. Some was contained in Electronic Medical Records documented by treating neurologists for years after the P3 trial ended in 2020. Some improvements in function were documented in videos. That RWE showed unprecedented trach-free survival benefits as of October 2026. In the 10-person EAP: 🔹5 yr survival = 100% EAP vs <20% ALS natural history 🔹5 yr median survival benefit = 7.5 yrs EAP vs 2.5 yrs ALS natural history 🔹5/10 people still alive, trach-free 🔹EAP's 51 patient treatment years ≈ patient treatment years in short 28-wk P3 trial 🔹Median not yet reached by those in the treatment arm in the trial and EAP (N-N) where 4/6 are still alive (range 7.5 - 9.5 yrs) with 81% lower risk of trach or death Although OS and trach-free survival were pre-specified, the data were not yet ripe when the 28-week P3 trial ended. Now it is. The FDA has never considered this #RWE. The data confirm the AdComm testimony of renowned PI experts, treating neurologists and the post hoc findings of the 28-week trial. The FDA approves cancer therapies based on 6 months of additional survival. NurOwn has extended survival by FIVE YEARS in a 100% terminal rare disease with no disease modifying treatments. @FDA_KyleD @FDADrugs The Rare Disease Framework will take years to implement -- years people with ALS don't have. Thus, we are asking the FDA and CBER to use its regulatory flexibility, act urgently, and grant accelerated approval of #NurOwn. #EndALS #ALS #MND #rarediseases #clinicaltrials #CitizenScientists #PatientAdvocacy #neurology #neurologist @DorisMatsui @Bilirakis @RepGuthrie @RepJohnJoyce @RepGregMurphy @RepMGriffith @RepMMM @RepAndyHarrisMD @RepSchakowsky @HouseCommerce @EnergyCommerce @GOPHELP @SenateAging @SenateAgingDems @ChrisCoons @lisamurkowski @SenBillCassidy @SenRickScott @SenRonJohnson @SenatorWicker @ChuckGrassley @LeaderJohnThune @SenWhitehouse @RandPaul @SenJohnBarrasso @DrHMelamed @houmanhemmati @CNBC @nyahphengsitthy @hmac8 @biospace @JefAkst @AP_FDAwriter @SquawkCNBC @BeckyQuick

No mother should have to bury a child. King Nazir had an ultra-rare form of pediatric #ALS caused by a de novo mutation in the #SPTLC2 gene. @NIHDirector_Jay please help advance therapy development for these pediatric and juvenile patients. @eperlste #RareDiseases

"Catch up" #CBER? For 500+ days, @FDADrugs has possessed unprecedented survival data in #NurOwn Citizens' Petition but has failed to respond. Real-world Evidence from EAP: • 100% 5 yr trach-free survival vs <20% in natural history • >7.5 yr median trach-free survival vs 2.5 yrs natural history. It's been 500+ days on @FDA's 180-day regulatory deadline to respond to the citizen's petition -- at the cost of 8k+ lives. @FDA_KyleD We need action, not speeches. #EndALS #rarediseases @houmanhemmati @LeaderJohnThune @ChuckGrassley @SenatorWicker @RonJohnsonWI @SenGillibrand @SenRickScott @RandPaul @Bilirakis @DorisMatsui @RepGuthrie @RepMMM @RepJohnJoyce @RepGregMurphy @RepDonBacon @RepMGriffith @RepSchakowsky

If #CBER wants to honor the new framework, it can start by paying attention to the unprecedented survival data in the #NurOwn citizens' petition -- the ultimate clinically meaningful endpoint -- and the 2002 Public Comments at the AdCom explaining #ALS patients' tolerance for risk. It's been over 500 days on a 180-day regulatory deadine. In that time, more than 8,000 Americans with ALS have #DiedWaiting. #EndALS #RareDiseases @FDAdrugs @houmanhemmati @LeaderJohnThune @ChuckGrassley @SenatorWicker @RonJohnsonWI @SenGillibrand @SenRickScott @RandPaul