Rare Diseases Today—February 12, 2026

This week’s Rare diseases update covers regulatory actions, clinical designations, access programs, policy benchmarking, and partnerships across key markets.

In Today’s Newsletter

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❌ FDA CRL for RGX-121 in Hunter syndrome (Regenxbio) [1] [US • 09 Feb 2026]

https://www.fiercebiotech.com/biotech/fda-rejects-regenxbio-hunter-syndrome-gene-therapy-recently-hit-hold-unrelated-brain-tumor
Context: Decision followed a PDUFA delay and a separate clinical hold tied to an RGX-111 tumor event, which FDA did not cite as a rejection reason; sponsor plans resubmission with longer-term data.
Key point: FDA rejected RGX-121, flagging patient-population definition, natural history control, and use of heparan sulfate as a surrogate endpoint (accelerated approval path not granted).
Implication: May influence prescriber choice and payer reviews pending full data.

🧬 Rilzabrutinib lands FDA BTD and Japan ODD in wAIHA (Sanofi) [2] [09 Feb 2026]

https://www.globenewswire.com/news-release/2026/02/09/3234232/0/en/Press-Release-Sanofi-s-rilzabrutinib-designated-breakthrough-therapy-in-the-US-and-orphan-drug-in-Japan-for-the-treatment-of-warm-autoimmune-hemolytic-anemia.html
Context: Based on LUMINA 2 phase 2b data (ongoing); LUMINA 3 phase 3 is underway versus placebo (endpoints not specified).
Key point: FDA granted Breakthrough Therapy, and Japan granted orphan designation, for rilzabrutinib in wAIHA.
Implication: May influence prescriber choice and payer reviews pending full data.

📦 Exclusive US distribution for Redemplo (plozasiran) in FCS (Vanscoy Rare Pharmacy) [3] [US • 10 Feb 2026]

https://www.newswise.com/articles/vanscoy-rare-pharmacy-selected-as-exclusive-distribution-pharmacy-for-redemplo-plozasiran-for-adults-with-familial-chylomicronemia-syndrome-fcs
Context: siRNA therapy indicated as adjunct to diet to reduce triglycerides in FCS; agreement includes end-to-end patient support.
Key point: Vanscoy Rare Pharmacy selected as the exclusive specialty pharmacy for Redemplo (plozasiran) for adults with FCS.
Implication: May expand screening, initiation, and follow-up at scale.

🏛️ NORD grades states on rare-disease access, national “B” [4] [US • 09 Feb 2026]

https://www.prnewswire.com/news-releases/national-organization-for-rare-disorders-ranks-all-50-states-on-rare-disease-policies-302682833.html
Context: Only Colorado, Delaware, Maine, and New Jersey earned overall A grades; 19 states still lack RDACs.
Key point: 2025 State Report Card shows improvements in telehealth and affordability but persistent gaps in medical nutrition and insurance safeguards.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🧫 ENCell’s EN001 gets FDA ODD in DMD (second ODD) [5] [US • 06 Feb 2026]

https://biz.chosun.com/en/en-science/2026/02/06/4VMLHJQIVRFGPBZ7BEWEZHLKNI/
Context: Platform aims to modulate inflammation and support tissue repair; benefits of ODD include potential 7-year exclusivity, tax credits.
Key point: EN001, an allogeneic WJ-MSC therapy, received FDA orphan designation for DMD, following prior ODD in CMT.
Implication: Signals pipeline investment and modality expansion.

🧪 Zydus desidustat earns FDA ODD in sickle cell disease [6] [US • 06 Feb 2026]

https://scanx.trade/stock-market-news/stocks/zydus-lifesciences-receives-tentative-usfda-approval-for-dapagliflozin-tablets/31742221
Context: Orphan status may confer 7-year exclusivity upon approval and development incentives; specifics of program not stated.
Key point: FDA granted orphan drug designation to desidustat for sickle cell disease.
Implication: Signals pipeline investment and modality expansion.

🇰🇷 Free 12-month Wegovy program for rare-disease obesity in Korea [7] [KR • 10 Feb 2026]

https://www.koreabiomed.com/news/articleView.html?idxno=30598
Context: Collaboration among Korean Organization for Rare Diseases, Novo Nordisk Korea, Community Chest of Korea, and KSSO; selection via clinician recommendation and review.
Key point: Program will supply ~300 eligible patients with 12 months of Wegovy pens, targeting obesity with rare diseases and low-income groups.
Implication: May expand screening, initiation, and follow-up at scale.

🤝 THX Pharma–Biocodex licensing for Batten-1 and TX01 (up to €173m) [8] [FR • 10 Feb 2026]

https://www.actusnews.com/en/amp/theranexus/pr/2026/02/10/thx-pharma-and-biocodex-announce-a-strategic-licensing-agreement-covering-three-rare-diseases
Context: Deal includes €12m upfront, up to €161m in milestones, and tiered double-digit royalties; THX leads clinical development with Biocodex support.
Key point: Biocodex licensed Batten-1 globally and TX01 for US/Canada, covering Batten disease, Gaucher, and Niemann-Pick type C.
Implication: Signals pipeline investment and modality expansion.

Why it matters

FDA’s CRL for RGX-121 underscores rising scrutiny of surrogate endpoints and natural history controls in ultra-rare CNS gene therapies.
Rilzabrutinib’s designations could accelerate options in wAIHA, where targeted therapies are limited.
Centralized distribution for Redemplo may streamline access and monitoring in FCS.
Policy variation by state continues to shape real-world access for 30 million Americans with rare diseases.
New ODDs and BD deals signal continued investment in rare neuromuscular and lysosomal disorders.

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FAQ

What did FDA cite in Regenxbio’s CRL for RGX-121?

FDA questioned patient-population definition, natural history control, and the surrogate biomarker heparan sulfate. Safety issues from RGX-111 were acknowledged but not cited as reasons for rejection [1].

Which data back Sanofi’s rilzabrutinib designations in wAIHA?

Preliminary data from the ongoing LUMINA 2 phase 2b study supported FDA BTD and Japan ODD, with the LUMINA 3 phase 3 now enrolling; endpoints not specified in the release [2].

What does Vanscoy’s Redemplo role cover?

Vanscoy Rare Pharmacy is the exclusive US specialty pharmacy for Redemplo (plozasiran) in adult FCS, providing start-to-finish patient support alongside dispensing [3].

Which states earned overall A grades in NORD’s report?

Colorado, Delaware, Maine, and New Jersey earned A grades. Nineteen states still lack RDACs, indicating uneven patient representation in policy [4].

What is ENCell’s EN001 and its ODD status?

EN001 is an allogeneic umbilical cord-derived MSC therapy. It now has FDA ODD for DMD, after a prior ODD in CMT, enabling incentives like potential 7-year exclusivity upon approval [5].

What did Zydus receive for desidustat?

FDA orphan drug designation in sickle cell disease, which may provide development incentives and post-approval exclusivity; program specifics were not stated [6].

Entities / Keywords

Regenxbio; RGX-121; RGX-111; Hunter syndrome; MPS II; heparan sulfate • Sanofi; Wayrilz; rilzabrutinib; wAIHA; LUMINA 2; LUMINA 3 • Vanscoy Rare Pharmacy; Redemplo; plozasiran; FCS; Arrowhead • NORD; State Report Card; RDACs • ENCell; EN001; DMD; CMT; WJ-MSC • Zydus Lifesciences; desidustat; sickle cell disease • Novo Nordisk Korea; Wegovy; KSSO; Korean Organization for Rare Diseases • THX Pharma (Theranexus); Biocodex; Batten-1; TX01; Gaucher; Niemann-Pick type C.