Rare Disease Today—January 22, 2026

This week’s updates focus on significant regulatory approvals, orphan drug designations, and clinical progress for rare diseases. Notable developments include breakthroughs in treatments for serious conditions and the expansion of access to critical therapies.

In Today’s Newsletter

Dive deeper

✨ Sobi’s Aspaveli approved for C3G and IC-MPGN in Europe [1] [EU • 16 Jan 2026]

https://www.sobi.com/en/press-releases/sobi-receives-european-commission-approval-aspavelir-pegcetacoplan-treatment-c3g-and-primary-ic-mpgn-2418695
Context: European Commission approves Aspaveli for treating C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) for patients aged 12+ years.
Key point: Aspaveli is the first therapy approved for these conditions in adolescent patients, offering hope for kidney disease patients with limited options.
Implication: Represents a significant advancement for rare kidney disease treatments, with potential to expand into new markets.

🧬 Rein Therapeutics receives Orphan Drug Designation for LTI-03 in IPF [2] [EU • 20 Jan 2026]

https://www.manilatimes.net/2026/01/20/tmt-newswire/globenewswire/rein-therapeutics-receives-orphan-drug-designation-from-european-medicines-agency-for-lead-drug-candidate-in-idiopathic-pulmonary-fibrosis/2261804
Context: EMA grants Orphan Drug status for LTI-03, aiming to preserve lung function in IPF patients.
Key point: This designation supports the development of new treatments for a progressive, rare lung disease with limited therapeutic options.
Implication: Rein’s breakthrough approach could address a critical unmet need in the IPF treatment landscape.

🧪 FDA grants Orphan Drug Designation to Atossa’s (Z)-Endoxifen in DMD [3] [US • 19 Jan 2026]

https://www.biospace.com/press-releases/atossa-therapeutics-receives-fda-orphan-drug-designation-for-z-endoxifen-for-the-treatment-of-duchenne-muscular-dystrophy
Context: (Z)-Endoxifen gains Orphan Drug Designation for treating Duchenne Muscular Dystrophy.
Key point: This rare pediatric neuromuscular disease has limited treatment options, and (Z)-endoxifen offers hope for better outcomes.
Implication: Could significantly impact the DMD treatment pipeline with potential new therapies.

🧬 BPGbio’s BPM31510 gets FDA Orphan Drug Designation for Primary CoQ10 Deficiency [4] [US • 20 Jan 2026]

https://sg.finance.yahoo.com/news/bpgbio-receives-fda-orphan-drug-120000379.html
Context: BPM31510 receives FDA Orphan Drug status for treating Primary CoQ10 Deficiency, a mitochondrial disorder.
Key point: The therapy is designed to address a rare and life-threatening condition with few treatment options.
Implication: The designation paves the way for expedited development and regulatory support for a potential breakthrough therapy.

🧴 Adolore BioTherapeutics receives Orphan Drug Designation for Kv7-activating therapy in Erythromelalgia [5] [US • 15 Jan 2026]

https://finance.yahoo.com/news/adolore-biotherapeutics-announces-fda-orphan-224000981.html
Context: Adolore’s gene therapy targeting Kv7 channels for Erythromelalgia receives Orphan Drug status.
Key point: The therapy aims to offer a non-opioid solution for severe chronic pain in EM patients.
Implication: This breakthrough could offer significant relief for EM patients suffering from debilitating pain.

💉 Genethon signs licensing agreement with AskBio for Pompe Disease gene therapy [6] [EU • 14 Jan 2026]

https://www.tanabe-pharma.com/en/news/rel_260115/main/0/link/e_rel_260115.pdf
Context: Genethon partners with AskBio to develop gene therapy for Pompe Disease, a rare genetic disorder.
Key point: The gene therapy aims to address the severe muscle and respiratory dysfunctions associated with the disease.
Implication: This partnership brings new hope for Pompe Disease patients and accelerates clinical development.

🔬 Tanabe Pharma’s MT-7117 shows positive results in EPP and XLP [7] [EU • 14 Jan 2026]

https://www.prnewswire.com/news-releases/alfasigma-secures-exclusive-rights-from-innovative-molecules-to-parenteral-adibelivir-for-treatment-of-hsv-encephalitis-an-ultra-rare-disease-302661452.html
Context: Tanabe’s MT-7117 shows favorable efficacy in treating Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP).
Key point: This selective melanocortin receptor agonist offers a potential new treatment for these rare and painful skin disorders.
Implication: The positive data could lead to the development of a much-needed treatment for EPP and XLP patients.

💊 Alfasigma secures exclusive rights to Adibelivir for HSV Encephalitis [8] [EU • 14 Jan 2026]

https://www.prnewswire.com/news-releases/alfasigma-secures-exclusive-rights-from-innovative-molecules-to-parenteral-adibelivir-for-treatment-of-hsv-encephalitis-an-ultra-rare-disease-302661452.html
Context: Alfasigma acquires worldwide rights to Adibelivir, targeting HSV encephalitis, an ultra-rare neurological condition.
Key point: The drug has shown promise in improving viral control and clinical outcomes in this life-threatening disease.
Implication: This could represent a major advancement in treating HSV encephalitis, addressing a critical unmet need.

🧠 Isturisa (osilodrostat) now available in Canada for Cushing’s Disease [9] [CA • 20 Jan 2026]

https://cushingsdiseasenews.com/news/isturisa-now-available-canada-adults-cushings-disease/
Context: Isturisa is now approved in Canada for treating adults with Cushing’s disease when surgery isn’t an option.
Key point: This approval expands the treatment landscape for this rare and debilitating condition.
Implication: Patients with persistent high cortisol levels now have access to a new therapeutic option in Canada.

Why it matters

The approval of Aspaveli marks a significant milestone for rare kidney diseases like C3G and IC-MPGN, offering new hope for patients in Europe.
Rein Therapeutics’ Orphan Drug Designation for IPF signals progress in addressing the unmet need for pulmonary fibrosis treatments.
FDA’s Orphan Drug Designations for therapies like (Z)-Endoxifen and BPM31510 are significant steps toward advancing treatments for rare neuromuscular and mitochondrial diseases.
Adolore’s approach to Erythromelalgia offers a novel, opioid-free pain relief option for patients with chronic pain conditions.

🚀 Accelerate your success. Contact us now

📂 Explore our case studies. See examples of our work.

💡 Read our insights. Learn from our latest reports and analysis

🎬 Watch on YouTube. Subscribe and never miss a video.

🧰 See our full range of services. Discover how we can help you.

📚 View the full Rare Disease archive on our research hub page

FAQ

What is Aspaveli approved for?

Aspaveli is approved for treating C3G and primary IC-MPGN in Europe, offering a first-line treatment for these rare kidney conditions [1].

What is Rein’s LTI-03 designed to treat?

LTI-03 is a treatment for idiopathic pulmonary fibrosis (IPF), a rare lung disease with limited treatment options [2].

What is the significance of Adolore’s Kv7-activating therapy?

Adolore’s therapy targets Kv7 channels to address the severe chronic pain in Erythromelalgia without the need for opioids [5].

How is Tanabe Pharma’s MT-7117 beneficial?

MT-7117 is a treatment for Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP), offering significant pain relief and skin protection [7].

Entities / Keywords

Sobi, Aspaveli, C3G, IC-MPGN, Rein Therapeutics, LTI-03, IPF, Atossa Therapeutics, (Z)-Endoxifen, Duchenne Muscular Dystrophy, BPGbio, BPM31510, Primary CoQ10 Deficiency, Adolore BioTherapeutics, Erythromelalgia, Genethon, Pompe Disease, Tanabe Pharma, MT-7117, Erythropoietic Protoporphyria, X-Linked Protoporphyria, Alfasigma, Adibelivir, HSV Encephalitis, Recordati, Isturisa, Cushing’s Disease.