This biweekly Rare diseases video recap highlights regulatory decisions, orphan designations, clinical development progress, and label expansions across multiple global markets.
🎯 Watch Our Video Summary Capturing Rare Disease News from the Last Two Weeks
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- Weeks 26 February – 4 March 2026
- Week 5 –11 March 2026
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Top Stories Covered In This Video
Chapters
0:00 Introduction
0:08 Soligenix SGX945 wins EMA orphan opinion in Behçet’s disease
0:41 Sanofi rilzabrutinib gets Japan orphan designation for IgG4-RD
1:09 Meiji Seika Pharma morcamilast secures EU orphan status for PPP
1:38 Chiesi expands JUXTAPID to pediatric HoFH in the US
2:04 Elfabrio gets EU every-4-weeks dosing option for stable Fabry patients
2:29 Sangamo moves ST-920 BLA forward in Fabry disease
2:59 Koselugo approved in Canada for adults with NF1 plexiform neurofibromas
3:24 Eledon tegoprubart gains FDA orphan tag in liver transplantation
3:51 How to reach us
Transcript
Welcome to the latest edition of Rare Diseases Updates, covering breakthroughs in the past two weeks. Brought to you by LucidQuest.
Soligenix received a positive opinion from the European Medicines Agency’s Committee for Orphan Medicinal Products for SGX945 in Behçet’s disease. SGX945 contains dusquetide and was submitted following Phase 2a results indicating biological efficacy and safety in the condition. The positive recommendation supports orphan drug designation, with final ratification by the European Commission expected as the next regulatory step.
Sanofi announced that Japan’s Ministry of Health, Labour and Welfare granted orphan drug designation to rilzabrutinib for IgG4-related disease. Rilzabrutinib is an oral reversible covalent BTK inhibitor. The designation follows Phase 2 data and comes as Sanofi continues the Phase 3 RILIEF study evaluating the therapy in patients with this immune-mediated disorder.
Meiji Seika Pharma reported that the European Commission granted orphan medicinal product designation to morcamilast, also known as ME3183, for palmoplantar pustulosis. Morcamilast is an oral selective PDE4 inhibitor being developed for autoimmune diseases with significant unmet need, and the designation supports continued clinical development in this indication.
The FDA approved an expanded indication for JUXTAPID, also known as lomitapide, allowing use in children aged two years and older with homozygous familial hypercholesterolemia. The decision was supported by results from the Phase 3 APH-19 open-label multicenter study involving pediatric participants between five and seventeen years of age.
In Europe, the European Commission approved an additional dosing regimen for Elfabrio, or pegunigalsidase alfa, in adults with Fabry disease who are stable on enzyme replacement therapy. The updated label allows a two milligram per kilogram infusion every four weeks, based on results from the BRIGHT open-label switch-over study and its extension.
Sangamo Therapeutics advanced its rolling biologics license application submission to the FDA for ST-920, also known as isaralgagene civaparvovec, for Fabry disease. The company reported submission of both preclinical and clinical modules and noted alignment with the FDA on using the fifty-two week eGFR slope as a potential endpoint for accelerated approval review.
Health Canada approved Koselugo, or selumetinib, for adults with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas. The decision was supported by results from the Phase 3 KOMET trial, which AstraZeneca described as the largest and only global placebo-controlled study conducted in this adult population.
Eledon Pharmaceuticals announced that the FDA granted orphan drug designation to tegoprubart for prevention of allograft rejection in liver transplantation. The therapy had previously received orphan designation for pancreatic islet cell transplantation and amyotrophic lateral sclerosis, and the company cited kidney transplant clinical data alongside preclinical liver transplant research.
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Why it matters
- Rare disease regulatory momentum remains broad, across orphan designations, label expansions, dosing flexibility, and late-stage filing progress. [1][2][3][4][5][6][7][8]
- Fabry disease was especially active this week, with both an EU dosing update for Elfabrio and US filing progress for Sangamo’s ST-920. [5][6]
- Several updates focus on access and practical treatment burden, not only new molecules, especially JUXTAPID in pediatric HoFH and Elfabrio every-4-weeks dosing. [4][5]
- Geographic spread matters, with decisions across the EU, Japan, the US, and Canada highlighting multi-region rare disease competition. [1][2][3][4][5][7][8]
- Orphan designations for SGX945, rilzabrutinib, morcamilast, and tegoprubart reinforce continued investment in smaller, high-unmet-need indications. [1][2][3][8]
🗓️ Explore weekly details and sources
- Weeks 26 February – 4 March 2026
- Week 5 –11 March 2026
📚 Find your one-stop page for the full Rare Disease archive.
FAQ
What changed for JUXTAPID (lomitapide) in HoFH?
FDA expanded JUXTAPID’s indication to include children aged 2 years and older with homozygous familial hypercholesterolemia. Chiesi said the decision was supported by APH-19 pediatric study data. [4]
What is the regulatory significance of Soligenix’s SGX945 update?
The EMA orphan committee issued a positive opinion for SGX945 in Behçet’s disease. The release states the next step is European Commission ratification, so this is not yet the final EC designation notice. [1]
Why is Sanofi’s rilzabrutinib designation notable in IgG4-RD?
Japan granted orphan drug designation based on Phase 2 data, while Sanofi is also running the Phase 3 RILIEF study in IgG4-related disease. It is described as the third global orphan designation for this indication. [2]
What does the Elfabrio every-4-weeks approval mean in practice?
The EC approved a less frequent dosing schedule for eligible adults with Fabry disease who are stable on ERT. The main practical change is a longer infusion interval, from every two weeks to every four weeks. [5]
How far along is Sangamo’s ST-920 filing?
Sangamo said the preclinical and clinical modules of the rolling BLA have been submitted to FDA, following initiation of the rolling submission in December 2025. The therapy remains investigational. [6]
What is new about Koselugo in Canada?
Health Canada approved selumetinib for adults with NF1 and symptomatic, inoperable plexiform neurofibromas. This extends availability beyond pediatric use in Canada, according to AstraZeneca’s release. [7]
Entities / Keywords
Soligenix, SGX945, dusquetide, Behçet’s disease, EMA, COMP, orphan drug designation
Sanofi, rilzabrutinib, BTK inhibitor, IgG4-related disease, IgG4-RD, MHLW, Japan orphan drug designation
Meiji Seika Pharma, morcamilast, ME3183, PDE4 inhibitor, palmoplantar pustulosis, PPP, European Commission orphan designation
Chiesi Global Rare Diseases, JUXTAPID, lomitapide, homozygous familial hypercholesterolemia, HoFH, pediatric approval, FDA
Chiesi Global Rare Diseases, Protalix BioTherapeutics, Elfabrio, pegunigalsidase alfa, Fabry disease, every-4-weeks dosing, European Commission
Sangamo Therapeutics, ST-920, isaralgagene civaparvovec, Fabry disease, rolling BLA, accelerated approval, eGFR slope
Alexion, AstraZeneca Rare Disease, Koselugo, selumetinib, neurofibromatosis type 1, NF1, plexiform neurofibromas, Health Canada
Eledon Pharmaceuticals, tegoprubart, liver transplantation, allograft rejection, orphan drug designation, FDA
