About Larimar Therapeutics Larimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. Larimar’s lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's ataxia. Nomlabofusp program granted Breakthrough Therapy Designation for the treatment of adults and children with FA based on FDA’s review of available clinical... (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to nomlabofusp, a frataxin (FXN) protein replacement therapy with disease modifying potential, for the treatment of adults and children with Friedreich’s ataxia (FA). Additionally, after a recent Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program meeting with FDA, the Company announced continued alignment with the FDA to consider the use of skin FXN as a novel surrogate endpoint reasonably likely to predict clinical benefit to support a planned Biologics License Application (BLA) submission seeking accelerated approval. The Breakthrough Therapy Designation request for nomlabofusp included preliminary clinical data from the OL study demonstrating increases in skin FXN to levels expected in asymptomatic carriers and consistent directional improvement across four key clinical outcomes including modified Friedreich Ataxia Rating Scale (mFARS) score, FARS-Activities of Daily Living (ADL), 9 Hole Peg Test (9-HPT), and Modified Fatigue Impact Scale (MFIS) after 1-year on treatment. These findings reinforce the potential of nomlabofusp to improve FA’s disease course relative to a worsening observed in a reference g BALA CYNWYD, Pa., Dec. 18, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare...

Original title: “Larimar Therapeutics Announces FDA Breakthrough Therapy Designation for Nomlabofusp in FA and Reiterates Planned BLA Submission in June 2026”

There are currently no FDA-approved treatments for the estimated more than 30,000 diagnosed patients with microcystic LMs in the United States. ... Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company ... There are currently no FDA-approved treatments for the estimated more than 30,000 diagnosed patients with microcystic LMs in the United States. ... Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat participants suffering from serious, rare skin diseases and vascular malformations for which there are no FDA-approved therapies. Primary endpoint met with statistically significant improvement (mean change of +2.13; p (Palvella or “the Company”), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat participants suffering from serious, rare skin diseases and vascular malformations for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced positive topline results from the Company’s Phase 3 SELVA study of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs). If approved, QTORIN™ rapamycin would be the first approved therapy for patients with microcystic LMs. SELVA is a Phase 3, single-arm, baseline-controlled clinical trial evaluating once-daily QTORIN™ rapamycin in individuals aged ≥ 3 years with microcystic LMs. Of the 51 participants enrolled, 50 initiated treatment, including 49 participants aged ≥ 6 years and 1 participant in the exploratory 3- to 5-year-old cohort.

Original title: “Palvella Therapeutics Announces Positive Topline Results from Phase 3 SELVA Clinical Study of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) in Microcystic Lymphatic Malformations”

The FDA expects a flood of applications for a new "plausible mechanism pathway" for approval of bespoke gene-editing treatments. "Plausible mechanism pathway" is to make it easier for individual gene-editing success stories like Baby KJ to be produced at a vastly wider scale. The new pathway has been greeted enthusiastically by academics, companies, and patient groups who see it as the only way to usher in gene editing-based treatments and other drugs for patients with unique mutations or mutations too rare to interest pharma. FDA Commissioner Marty Makary helped draft the new approach on rare diseases.Andrew Harnik/Getty Images

Original title: “FDA unveils new pathway for rare disease treatments | STAT”

The FDA expects a flood of applications for a new "plausible mechanism pathway" for approval of bespoke gene-editing treatments. "Plausible mechanism pathway" is to make it easier for individual gene-editing success stories like Baby KJ to be produced at a vastly wider scale. The new pathway has been greeted enthusiastically by academics, companies, and patient groups who see it as the only way to usher in gene editing-based treatments and other drugs for patients with unique mutations or mutations too rare to interest pharma. FDA Commissioner Marty Makary helped draft the new approach on rare diseases.Andrew Harnik/Getty Images

Original title: “FDA unveils new pathway for rare disease treatments | STAT”

Over the next 18 months, the ... phase 1 clinical trial and preparing the investigational new drug application for submission to the FDA. Blue Gen Therapeutics Foundation also licensed ADVM-082, an intravitreal gene therapy that encodes a functional CNGB3 gene for the treatment of achromatopsia, another rare disease that leads ... Over the next 18 months, the Children’s Hospital Los Angeles and the Blue Gen Therapeutics Foundation will conduct the work to prepare for clinical trials for BGTF-027, including developing a protocol for a phase 1 clinical trial and preparing the investigational new drug application for submission to the FDA. Blue Gen Therapeutics Foundation also licensed ADVM-082, an intravitreal gene therapy that encodes a functional CNGB3 gene for the treatment of achromatopsia, another rare disease that leads to vision that has no color. The Vision Center at Children’s Hospital Los Angeles is developing a protocol for a phase 1 clinical trial for the first gene therapy for boys with blue cone monochromacy, which impairs color vision and severely affects visual function. ... received a $4.7 million grant from the California Institute for Regenerative Medicine to study a gene therapy to treat blue cone monochromacy (BCM), a rare, inherited vision disorder linked to the X chromosome that occurs in boys. Currently, there is no treatment for this disease. Aaron Nagiel, M.D., Ph.D., an investigator conducting ophthalmology and neuroscience research at Children’s Hospital Los Angeles, will lead efforts on research of a gene therapy that aims to slow the progression of disease and restore vision loss. Nagiel is also an assistant professor of Clinical Ophthalmology at Keck School of Medicine of USC. licensed to Blue Gen Therapeutics Foundation (BGTF), a nonprofit organization developing gene therapies for rare inherited retinal diseases. Blue Gen Therapeutics Foundation was co-founded by John Cavitt, who has three children affected by blue cone monochromacy. BGTF-027 is an intravitreal gene therapy that encodes a functional copy of the long-wavelength-sensitive opsin protein for the treatment of blue cone monochromacy.

Original title: “Gene Therapy for Rare Eye Disease to Advance to Human Trial | Managed Healthcare Executive”

QUASAR long‑term extension results highlight durable clinical and endoscopic outcomes

Original title: “Tremfya data show sustained remission in ulcerative colitis through 3 years”

MHRA authorises medicine for patients aged 12 and over with repeated flare‑ups

Original title: “Brensocatib becomes first licensed treatment for non‑cystic fibrosis bronchiectasis”

Bysanti is based on iloperidone, an active metabolite of a compound that forms the core of Fanapt, another drug by Vanda Pharmaceuticals.

Original title: “Vanda Rebounds From Jet Lag Rejection With FDA Nod for Bipolar, Schizophrenia Drug”

PDUFA action date set for August 23, 2026 If approved, DTX401 will be the first treatment to address the underlying cause of GDSIa NOVATO, Calif., Feb. ... The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. (NASDAQ: RARE) today announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the Biologics License Application (BLA) seeking approval of DTX401 AAV gene therapy (pariglasgene brecaparvovec) for the treatment of Glycogen Storage Disease Type Ia (GSDIa). Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, the ability of the company to successfully develop DTX401, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to adverse side effects, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, the company’s limited experience in operating its own manufacturing facility, the ability of the company and its third party

Original title: “Ultragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)”

ITF Therapeutics was established in January 2024 as the United States affiliate of Italfarmaco Group to develop and commercialize treatments for rare diseases. The company combines end-to-end execution with close listening to learn directly from advocates, researchers, clinicians, and families, ... ITF Therapeutics was established in January 2024 as the United States affiliate of Italfarmaco Group to develop and commercialize treatments for rare diseases. The company combines end-to-end execution with close listening to learn directly from advocates, researchers, clinicians, and families, ensuring their perspectives help shape the design of innovative and potentially life-changing solutions. Presenting ten abstracts including observations on long-term safety, disease progression, and decline in muscle contractile area with givinostat · Encore oral presentation also highlights analyses from the givinostat clinical development program on weight-based flexible dosing · CONCORD, Mass., Feb. 23, 2026 /PRNewswire/ -- ITF Therapeutics LLC, the U.S. rare disease affiliate of Italfarmaco, today announced that one oral and nine poster presentations have been accepted at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference to be held March 8-11, 2026, in Orlando, Florida. The company is a leader in pharmaceutical research, product development, production, and commercialization with proven success in many therapeutic areas including immuno-oncology, gynecology, neurology, cardiovascular disease, and rare diseases. Italfarmaco's rare disease unit includes programs in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis, and polycythemia vera. For more information visit ... DUVYZAT is a histone deacetylase inhibitor indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. This is not an official event of the 2026 MDA Clinical and Scientific Conference. This event is not sponsored, endorsed, or accredited by MDA. ... DUVYZAT is a U.S. FDA-approved histone deacetylase (HDAC) inhibitor indicated for the treatment of patients six years of age and older with Duchenne muscular dystrophy (DMD). The therapy was discovered through the research and development efforts of Italfarmaco in collaboration with Telethon and Duchenne Parent Project (Italy).

Original title: “ITF Therapeutics LLC Announces Multiple Data Presentations at 2026 MDA Clinical and Scientific Conference”

D3S-001 has received FDA breakthrough therapy and orphan drug designations in KRAS G12C-mutated NSCLC and CRC, respectively. The FDA has granted breakthrough therapy designation to D3S-001 for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non–small cell lung cancer (NSCLC) who have received prior chemotherapy and immunotherapy but have not been previously treated with a KRAS G12C inhibitor.1 · D3S-001 has also been granted orphan drug designation (ODD) for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic colorectal cancer (CRC). D3 Bio, Inc. announces FDA breakthrough therapy designation and orphan drug designation for D3S-001 for the treatment of patients with KRAS G12C-mutated cancers. News Release. D3 Bio. August 28, 2025. Accessed August 28, 2025. D3S-001 received FDA breakthrough therapy and orphan drug designations for KRAS G12C-mutated NSCLC and CRC, highlighting its potential in addressing unmet needs. "We are very pleased to receive both breakthrough therapy and orphan drug designations from the FDA for D3S-001, which highlights D3S-001's promising potential to address critical unmet needs in patients with KRAS G12C–mutated cancers," George Chen, founder and CEO of D3 Bio, stated in the news release.1 "These designations also recognize D3S-001's novel profile as a next-generation KRAS G12C inhibitor.

Original title: “D3S-001 Receives FDA Breakthrough Therapy, Orphan Drug Designations in Select KRAS G12C-Mutant Solid Tumors | OncLive”

Parkinson disease continues to ... therapies, potential disease-modifying strategies, biomarker refinement, and earlier diagnostic approaches. While major headlines often draw immediate attention, many ongoing studies may have an equal or greater impact on long-term treatment decision making. With a rapidly advancing research pipeline, it is challenging to narrow the field. Here, we highlight several of the most clinically meaningful ongoing Parkinson disease trials likely to ... Parkinson disease continues to evolve as an area of active investigation, with progress spanning symptomatic therapies, potential disease-modifying strategies, biomarker refinement, and earlier diagnostic approaches. While major headlines often draw immediate attention, many ongoing studies may have an equal or greater impact on long-term treatment decision making. With a rapidly advancing research pipeline, it is challenging to narrow the field. Here, we highlight several of the most clinically meaningful ongoing Parkinson disease trials likely to influence care as we move into 2026. As part of NeurologyLive®'s Year in Review, take a look at some of the top ongoing clinical trials in Parkinson disease that may impact care. The ACTIVATE trial examines BIA 28-6156 for GBA-PD, assessing its impact on MDS-UPDRS scores and other clinical measures, with promising phase 1 safety data. Gene therapy with AB-1005 in the REGENERATE-PD trial shows potential for improving Parkinson's symptoms, with prior phase 1b results indicating successful putamen coverage. The agent, which showed promise in a phase 1 study, is currently being assessed in the phase 2 REGENERATE-PD trial (NCT06285643), an 87-patient cohort study with trial sites in Germany, Poland, the United Kingdom, and the United States. In this randomized, double-blind, surgery-controlled study, investigators will use change in normalized good ON time per Hauser Diary over an 18-month treatment period as the primary outcome.5 · Currently, there are no FDA-approved therapies for PD that offer the same mechanism and target as AB-1005 does.

Original title: “Previewing Parkinson Disease Pipeline: Emerging Trials to Watch in 2026 | NeurologyLive - Clinical Neurology News and Neurology Expert Insights”

The agency could by late December approve a therapy that Roche sees as potentially becoming a new treatment standard for certain breast tumors.

Original title: “Roche gets FDA decision date on closely watched breast cancer drug”

Each year, the NeurologyLive® ... therapeutic pipeline, tracking late-stage clinical programs and regulatory milestones that have the potential to influence near-term clinical practice. As part of that effort, the team provides timely, in-depth coverage of FDA decisions that shape the evolving treatment landscape across neurologic disease states, aligning regulatory updates with expert insight on the underlying clinical trial ... Each year, the NeurologyLive® team closely monitors the neurologic therapeutic pipeline, tracking late-stage clinical programs and regulatory milestones that have the potential to influence near-term clinical practice. As part of that effort, the team provides timely, in-depth coverage of FDA decisions that shape the evolving treatment landscape across neurologic disease states, aligning regulatory updates with expert insight on the underlying clinical trial data. Explore anticipated FDA decisions on groundbreaking neurology drugs in 2026, shaping treatment options for various neurologic conditions. Looking ahead to the first half of 2026, several high-profile FDA decisions are expected across a range of neurologic conditions, spanning neurodegenerative disease, neuromuscular disorders, headache, epilepsy, and rare neurologic populations. These upcoming actions include anticipated approvals, regulatory verdicts on novel mechanisms, and decisions that could introduce new formulations, delivery strategies, or first-in-class options for patients with limited therapeutic alternatives. Tolebrutinib, a BTK inhibitor, shows promise in delaying disability progression in non-relapsing secondary progressive multiple sclerosis, with significant trial results supporting its potential approval. GTx-104, an injectable nimodipine formulation, demonstrated reduced hypotension and improved outcomes in aneurysmal subarachnoid hemorrhage, offering a reliable alternative to oral administration. Tavapadon targets D1-like receptors in Parkinson's disease, aiming to enhance motor control with fewer side effects compared to traditional dopaminergic therapies.

Original title: “Previewing Expected FDA Decisions in Neurology for 2026 | NeurologyLive - Clinical Neurology News and Neurology Expert Insights”

The session will focus on the latest ... III clinical trial, specifically highlighting the systemic disease-modifying capabilities of CORDStrom™ in patients with RDEB ... Unlike current standard-of-care options that are limited to topical wound treatments, CORDStrom™ is being developed as a systemic, disease-modifying therapy... The session will focus on the latest results from the MissionEB Phase III clinical trial, specifically highlighting the systemic disease-modifying capabilities of CORDStrom™ in patients with RDEB ... Unlike current standard-of-care options that are limited to topical wound treatments, CORDStrom™ is being developed as a systemic, disease-modifying therapy. Clinical investigators from the trial to share real-world experience treating RDEB patients with CORDStrom and present new data from the MissionEB clinical study Anna Martinez, MBBS, MRCP, FRCPCH - Principal investigator of the Mission EB Clinical trial. Dr. Martinez is the United Kingdom’s leading specialist in pediatric RDEB and is internationally recognized for her expertise in rare and complex skin disorders. Prof. Mark Lowdell, PhD, FRCPath FRSB - Chief Scientific Officer and Co-Founder of INmune Bio. Prof. Lowdell is also Professor of Cell and Tissue Therapy at University College London, where he has led a translational immunotherapy group since 1994.Registration Details INmune Bio Inc. is a publicly traded (NASDAQ: INMB), clinical-stage biotechnology company focused on developing treatments that target the innate immune system to fight disease. INmune Bio has three product platforms: (1) CORDStrom™, a proprietary pooled, allogeneic, human umbilical cord-derived mesenchymal Stromal/Stem cell (hucMSCs) platform that recently completed a blinded randomized trial in recessive dystrophic epidermolysis bullosa; (2) XPro™, a Dominant-Negative Tumor Necrosis Factor (DN-TNF) product platform designed to selectively neutralize soluble TNF, a key driver of inflammation and innate immune dysfunction; and (3) INKmune®, a cell-based medicine designed to prime a patient’s natural killer cells to eliminate minimal residual disease in patients with cancer.

Original title: “INmune Bio Announces Upcoming Webinar to Present New Clinical Data on CORDStrom™ for RDEB | The Manila Times”

Basque company becomes first authorised under new European SoHO rules

Original title: “Mikrobiomik secures approval for gut‑microbiota therapy for C difficile infection”

Review upcoming clinical trials with data readouts expected in the first half of 2026, providing updates relevant to ongoing research and clinical practice in neurology. The ADEPT-2 study assesses xanomeline/trospium for psychosis in Alzheimer's, potentially introducing a new class of therapies targeting muscarinic receptor agonism. The ELEVATE-PD trial examines IPX203 for Parkinson's, focusing on real-world efficacy and safety in patients with motor complications. REMODEL studies compare remibrutinib and teriflunomide in relapsing multiple sclerosis, with primary endpoints including annualized relapse rate and disability progression.SHOW MORE · Review upcoming clinical trials with data readouts expected in the first half of 2026, providing updates relevant to ongoing research and clinical practice in neurology. As the field of neurology advances, the early months of 2026 are anticipated to bring important clinical trial readouts that may influence research and care across multiple neurological conditions. These studies span a broad spectrum of disorders and aim to clarify disease mechanisms while evaluating emerging therapeutic approaches. Ranging from novel gene-based strategies to new pharmacologic interventions, the results may inform future treatment paradigms and clinical decision-making. At baseline, patients had a Clinical Dementia Rating-Sum of Boxes (CDR-SB) mean score, the primary end point, of 3.45 (± 1.70). On other secondary outcomes, such as Alzheimer’s Disease Assessment Scale – Cognitive Subscale, 13-item (ADAS-Cog13), patients had a baseline score of 27.75 (± 8.02). Recently, the FDA accepted an updated protocol for Annovis Bio’s · pivotal phase 3 AD study (NCT06709014) testing the therapeutic potential of its investigational agent buntanetap, an orally available small molecule, in AD. The study is designed to evaluate the primary end point of change in the Neuropsychiatric Inventory-Clinician: Hallucinations and Delusions score and the key secondary endpoint of Clinical Global Impression-Severity, with additional assessments on safety and tolerability compared with placebo. In the trial, participants aged 55 to 90 years with possible or probable AD disease will be randomly assigned to either an oral capsule of xanomeline/trospium or an oral capsule of placebo.4 Xanomeline/trospium, which is currently approved for schizophrenia in adults, has the potential to be the first treatment in a new class of pharmacologic therapies targeting agitation and psychosis based on muscarinic receptor agonism.5 Additional study results from the ADEPT program in psychosis associated with AD, including ADEPT-2, ADEPT-1 and ADEPT-4, are expected to be read out by the end of 2026.

Original title: “Key Neurology Trial Readouts to Watch in Early 2026 | NeurologyLive - Clinical Neurology News and Neurology Expert Insights”

Catch up on any of the neurology headlines you may have missed in January 2026, compiled into 1 place by the NeurologyLive® team. The coverage includes the latest FDA approvals, new designations, submissions, resubmissions, and clinical trial initiations and holds. Click the read more buttons for more details and information about each update. On January 5, 2026, the FDA approved ScinoPharm Taiwan’s glatiramer acetate (GA) injection as a treatment for relapsing multiple sclerosis (MS), making it the first complex injectable generic approval for this therapy.1 That guidance allows waiving traditional clinical bioequivalence trials if the generic demonstrates tight structural and compositional matching, as well as in vitro and in vivo functional similarity. On January 6, 2026, the FDA granted breakthrough therapy designation to Alkermes’ alixorexton, an investigational oral, selective orexin 2 receptor agonist, for the treatment of narcolepsy type 1 (NT1), using positive phase 1 and phase 2 data, including the phase 2 VIBRANCE-1 trial (NCT06358950), to support the decision. Months after the FDA issued a complete response letter (CRL) to the new drug application (NDA) for Sentynl Therapeutics’ copper histidinate, on January 12, 2026, the agency approved the agent as the first treatment for pediatric patients with Menkes disease, a rare genetic neurodegenerative disorder.3 Marketed as Zycubo, the therapy is a subcutaneous copper replacement treatment that delivers copper in a form designed to bypass impaired intestinal absorption and support systemic utilization of the mineral. "This milestone represents the culmination of decades of research into better understanding and ultimately finding an effective treatment for Menkes disease," Stephen Kaler, MD, a clinical genetics and genomics specialist, and professor of pediatrics at the Columbia University Medical Center, said in a statement.3 "Increased awareness of Menkes disease and rapid testing upon suspicion are critical, as beginning copper histidinate therapy in affected neonates has been shown to reduce symptoms and prolong life."

Original title: “FDA Action Update, January 2026: Approvals, Acceptance, and Clearance | NeurologyLive - Clinical Neurology News and Neurology Expert Insights”

Cytokinetics’ heart disease drug secured European approval. Elsewhere, Teva and Sanofi reported longer-term data from an experimental therapy targeting ulcerative colitis and Crohn’s disease.

Original title: “FDA accepts BMS protein degrader for review; Disc rare disease drug rejected”

The disagreement between Moderna and the FDA has reached a resolution just eight days after the biotech received a Refusal-to-File letter in response to its application for mRNA-1010. Moderna will now seek approval of the vaccine based on age.

Original title: “FDA Reverses Course on Moderna’s mRNA Flu Shot Application, Promising August Decision”