Rare Diseases Today—March 5, 2026

This is a weekly update in rare diseases, covering key regulatory actions, clinical program progress, and partnership activity. Updates include approvals and designations, pipeline financing and launches, and gene-therapy manufacturing and discovery collaborations.

In Today’s Newsletter

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🧬 Soligenix, SGX945 (dusquetide) orphan path in EU for Behçet’s [1] [EU • 26 Feb 2026]

https://www.biospace.com/press-releases/soligenix-receives-positive-opinion-from-the-european-medicines-agency-on-the-request-for-orphan-drug-designation-for-sgx945-for-the-treatment-of-behcets-disease
Context: EMA Committee for Orphan Medicinal Products (COMP) reviewed published Phase 2a results (biological efficacy and safety noted).
Key point: Soligenix said EMA COMP issued a positive opinion for orphan drug designation for dusquetide (SGX945) in Behçet’s disease, with European Commission ratification as the next step.
Implication: May influence prescriber choice and payer reviews pending full data.

🇯🇵 Sanofi, rilzabrutinib orphan designation in Japan for IgG4-RD [2] [Japan • 02 Mar 2026]

https://www.sanofi.com/en/media-room/press-releases/2026/2026-03-02-06-00-00-3247008
Context: Designation cited Phase 2 data (NCT04520451), and Sanofi noted an ongoing Phase 3 program (RILIEF, NCT07190196).
Key point: Japan’s MHLW granted orphan drug designation to rilzabrutinib (Sanofi, oral BTK inhibitor) for IgG4-related disease.
Implication: May influence prescriber choice and payer reviews pending full data.

💊 Chiesi, FDA expands JUXTAPID to pediatric HoFH (ages 2+) [8] [US • 03 Mar 2026]

https://www.globenewswire.com/news-release/2026/03/03/3248283/0/en/Chiesi-Global-Rare-Diseases-Announces-FDA-Approval-of-JUXTAPID-lomitapide-Capsules-for-Pediatric-Use-in-Homozygous-Familial-Hypercholesterolemia-HoFH.html
Context: Chiesi cited a Phase 3 open-label study (APH-19) in 43 pediatric participants ages 5–17, plus model-based analysis supporting dosing in children ages 2+.
Key point: FDA approved JUXTAPID (lomitapide) capsules for pediatric HoFH, expanding the US indication to children 2 years and older.
Implication: May influence prescriber choice and payer reviews pending full data.

🤝 Eton Pharmaceuticals adds HEMANGEOL US rights to rare disease portfolio [3] [US • 02 Mar 2026]

https://www.manilatimes.net/2026/03/02/tmt-newswire/globenewswire/eton-pharmaceuticals-expands-rare-disease-portfolio-through-acquisition-of-us-commercialization-rights-to-hemangeol-propranolol-hydrochloride-oral-solution/2291128
Context: HEMANGEOL is described as the only FDA-approved treatment for infantile hemangioma requiring systemic therapy, per the release.
Key point: Eton Pharmaceuticals in-licensed US commercialization rights to HEMANGEOL (propranolol hydrochloride oral solution) from Pierre Fabre, with commercialization beginning 01 May 2026.
Implication: Introduces competition that may affect pricing and formulary access.

🧪 Atrium Therapeutics launches with ~$270m for cardiac-targeted RNA medicines [4] [US • 27 Feb 2026]

https://investors.atriumtherapeutics.com/2026-02-27-Atrium-Therapeutics-Launches-with-Approximately-270-Million-to-Advance-Novel-RNA-Medicines-for-Rare-Genetic-Cardiomyopathies
Context: Atrium described lead programs ATR 1072 (PRKAG2 syndrome, IND filing targeted in 2H 2026) and ATR 1086 (PLN cardiomyopathy, IND submission targeted in 2027).
Key point: Atrium Therapeutics launched as an independent, publicly traded company to develop RNA therapeutics delivered to the heart, starting with two lead cardiomyopathy programs and ~$270m in cash and equivalents.
Implication: Signals pipeline investment and modality expansion.

🧫 Cure Rare Disease and LGMD2L Foundation fund ANO5 gene replacement therapy [5] [US • 03 Mar 2026]

https://www.biospace.com/press-releases/cure-rare-disease-and-lgmd2l-foundation-announce-multi-year-partnership-to-develop-gene-therapy-for-anoctamin-5-related-disease
Context: The release cites a $7.65m commitment and a path from design through preclinical work, manufacturing scale-up, and first-in-human trial readiness.
Key point: Cure Rare Disease and the LGMD2L Foundation announced a multi-year partnership to develop a gene replacement therapy for ANO5-related disease. Implication: Signals pipeline investment and modality expansion.

🏭 Progeria Research Foundation partners with Forge Biologics on SamPro-2 manufacturing [6] [US • 03 Mar 2026]

https://crisprmedicinenews.com/press-release-service/card/the-progeria-research-foundation-and-forge-biologics-announce-manufacturing-partnership-to-advance-g/
Context: SamPro-2 is described as an investigational AAV-delivered, base-editing approach intended to correct the Progeria mutation, with IND-enabling work supported.
Key point: The Progeria Research Foundation and Forge Biologics announced a manufacturing agreement to support development and manufacturing for SamPro-2 in Hutchinson-Gilford Progeria Syndrome.
Implication: Signals pipeline investment and modality expansion.

🇪🇺 Meiji Seika Pharma, morcamilast (ME3183) granted EU orphan designation for PPP [7] [EU • 03 Mar 2026]

https://www.biospace.com/press-releases/meiji-seika-pharmas-morcamilast-me3183-was-granted-orphan-medicinal-product-designation-by-the-european-commission-for-palmoplantar-pustulosis
Context: Morcamilast is described as an oral, selective PDE4 inhibitor, and the company referenced prior clinical work in psoriasis vulgaris (PPP program specifics not detailed).
Key point: Meiji Seika Pharma said the European Commission granted orphan medicinal product designation to morcamilast (ME3183) for palmoplantar pustulosis.
Implication: May influence prescriber choice and payer reviews pending full data.

🔬 Sinopia Biosciences and Ono Pharmaceutical target discovery in rare metabolic disorders [9] [US–Japan • 03 Mar 2026]

https://www.biospace.com/press-releases/sinopia-biosciences-announces-target-discovery-collaboration-with-ono-pharmaceutical-for-rare-metabolic-disorders
Context: The disorders are undisclosed, and financial terms beyond funding, upfront, and milestones were not disclosed.
Key point: Sinopia Biosciences and Ono Pharmaceutical entered a target discovery collaboration using Sinopia’s LEADS metabolomics-driven platform for rare metabolic disorders.
Implication: Signals pipeline investment and modality expansion.

Why it matters

Orphan designations in the EU and Japan (Soligenix, Sanofi, Meiji) can de-risk development plans and shape regulatory roadmaps in rare indications. [1], [2], [7]
The JUXTAPID label expansion underscores continued FDA willingness to extend rare disease therapies into younger pediatric populations when supported by evidence packages. [8]
Partnerships remain a primary execution lever in rare disease, especially where manufacturing and IND-enabling work are gating items (PRF–Forge, CRD–LGMD2L). [5], [6]
Portfolio and platform deals (Eton–HEMANGEOL, Sinopia–Ono, Atrium launch) signal sustained capital and BD focus despite long timelines in orphan markets. [3], [4], [9]

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FAQ

What is SGX945 (dusquetide), and what did EMA’s COMP do?

SGX945 is Soligenix’s program in Behçet’s disease, with dusquetide as the active ingredient. Soligenix reported EMA’s COMP issued a positive opinion on orphan designation, pending European Commission ratification. [1]

What does Japan’s orphan drug designation mean for rilzabrutinib in IgG4-RD?

Sanofi reported Japan’s MHLW granted orphan drug designation for rilzabrutinib in IgG4-related disease. Sanofi also referenced prior Phase 2 data and an ongoing Phase 3 study (RILIEF). [2]

What changed for JUXTAPID (lomitapide) in the US?

Chiesi said FDA approved JUXTAPID for pediatric HoFH, expanding the indication to children 2 years and older. The release referenced a pediatric Phase 3 study (APH-19) and supportive analyses. [8]

Who will commercialize HEMANGEOL in the US, and when?

Eton Pharmaceuticals said it in-licensed US commercialization rights from Pierre Fabre, and plans to begin US commercialization on 01 May 2026 via its Eton Cares program. [3]

What is SamPro-2, and what is Forge Biologics doing?

The Progeria Research Foundation described SamPro-2 as an investigational AAV gene therapy using a base-editing approach. Forge Biologics will support process development and manufacturing for IND-enabling studies. [6]

What is the Sinopia–Ono collaboration actually working on?

The companies described a target discovery collaboration for an undisclosed group of rare metabolic disorders, using Sinopia’s LEADS metabolomics-driven platform. Specific disorders and detailed financial terms were not disclosed. [9]

Entities / Keywords

Soligenix (SNGX), SGX945, dusquetide, Behçet’s disease, EMA COMP, orphan drug designation
Sanofi, rilzabrutinib, BTK inhibitor, IgG4-related disease (IgG4-RD), MHLW Japan, RILIEF (NCT07190196)
Eton Pharmaceuticals (ETON), HEMANGEOL (propranolol hydrochloride oral solution), Pierre Fabre, infantile hemangioma, Eton Cares
Atrium Therapeutics, RNA therapeutics, ATR 1072, PRKAG2 syndrome, ATR 1086, PLN cardiomyopathy
Cure Rare Disease (CRD), LGMD2L Foundation, ANO5, gene replacement therapy, AAV
Progeria Research Foundation (PRF), Forge Biologics, SamPro-2, Hutchinson-Gilford Progeria Syndrome, AAV, base editing
Meiji Seika Pharma, morcamilast (ME3183), PDE4 inhibitor, palmoplantar pustulosis (PPP), European Commission orphan designation
Chiesi Global Rare Diseases, JUXTAPID (lomitapide), homozygous familial hypercholesterolemia (HoFH), FDA approval, pediatric
Sinopia Biosciences, Ono Pharmaceutical, LEADS platform, metabolomics, rare metabolic disorders