Rare disease Archives

News and Trends 7 Sep 2022

Ionis presents positive study results at International Symposium on amyloidosis

Ionis Pharmaceuticals, Inc. has presented positive results from a planned 35-week interim analysis of the phase 3 NEURO-TTRansform study of Ionis and AstraZeneca’s eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). In the study, eplontersen demonstrated a statistically significant and clinically meaningful change from baseline for its co-primary and secondary efficacy endpoints compared to […]

September 7, 2022 - 4 minutesmins - By Jim Cornall

News and Trends 5 Sep 2022

Avacta’s drug targeting soft tissue sarcoma granted Orphan Drug Designation by FDA

A drug to treat soft tissue sarcoma has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). Clinical stage oncology drug company Avacta Group plc has produced a form of the generic chemotherapy, doxorubicin AVA6000. About Avacta’s drug The drug has been modified using the company’s pre|CISION technology and Affirmer […]

September 5, 2022 - 2 minutesmins - By Liza Laws

News and Trends 1 Sep 2022

Sanofi’s first treatment in the U.S. approved for adults and children with ASMD

A rare, inherited disease that affects the body’s ability to metabolize fat, cholesterol and lipids within cells can now be treated by Xenpozyme, a drug approved by the U.S. Food and Drug Administration (FDA). Adults and children with non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) can now take healthcare company Sanofi’s Xenpozyme […]

September 1, 2022 - 4 minutesmins - By Liza Laws

News and Trends 31 Aug 2022

Emactuzumab designated as an orphan medicinal product for TGCT in Europe

SynOx Therapeutics Limited says its novel monoclonal antibody, emactuzumab, in development for the treatment of tenosynovial giant cell tumor (TGCT) and other diseases, has been designated as an orphan medicinal product for both localized and diffuse types of the disease by the European Medicines Agency (EMA). No systemic treatments have been approved for TGCT in […]

August 31, 2022 - 4 minutesmins - By Jim Cornall

News and Trends 24 Aug 2022

Amgen announces top-line results from biosimilar phase 3 study

Amgen has announced positive top-line results from its DAHLIA study. This was a randomized, double-blind, active-controlled, two-period crossover phase 3 study evaluating the efficacy and safety of ABP 959, a biosimilar candidate to SOLIRIS (eculizumab), compared with SOLIRIS in adult patients with paroxysmal nocturnal hemoglobinuria (PNH). Amgen said the study met its primary endpoints, demonstrating […]

August 24, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 23 Aug 2022

Ocelot Bio receives FDA Orphan Drug Designation for lead candidate for treatment of hepatorenal syndrome

Ocelot Bio, Inc., a clinical-stage biopharmaceutical company, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead candidate OCE-205 for the treatment of hepatorenal syndrome. Ocelot Bio is currently enrolling patients in a phase 2 clinical trial of OCE-205 in hepatorenal syndrome with acute kidney injury (HRS-AKI), […]

August 23, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 22 Aug 2022

Vaderis emerges from stealth to start HTT trial

Vaderis Therapeutics AG, a clinical stage biotech company focused on developing treatments for rare diseases associated with vascular malformations, has emerged from stealth. The company has also started its INSIGHT proof-of-concept clinical trial in patients suffering from hereditary hemorrhagic telangiectasia (HHT), also known as Osler-Weber-Rendu syndrome. About HHT HHT is a disorder where some blood […]

August 22, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 19 Aug 2022

Emtora to use $16.9M grant to develop treatment for rare genetic disease

Emtora Biosciences, a Texas-based clinical-stage biopharmaceutical company, has been awarded a $16.9 million non-dilutive grant from the Cancer Prevention & Research Institute of Texas (CPRIT). The funding will be used to advance the clinical and product development of its lead program, eRapa, an encapsulated form of rapamycin, for the treatment of familial adenomatous polyposis (FAP). […]

August 19, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 19 Aug 2022

Aeglea BioTherapeutics’ arginine 1 deficiency treatment gets EMA ok

Aeglea BioTherapeutics, Inc. says its marketing authorization application (MAA) for pegzilarginase for the treatment of arginase 1 deficiency (ARG1-D) has been validated by the European Medicines Agency (EMA). The MAA was submitted by Immedica Pharma AB, Aeglea’s commercialization partner in Europe and the Middle East. Pegzilarginase is a novel, recombinant human arginase 1 enzyme that […]

August 19, 2022 - 3 minutesmins - By Jim Cornall

In Depth 22 Jul 2022

The coming of age story of RNAi technology

The first RNA interference (RNAi) therapy was approved in 2018, after 20 years in development. What can we expect to see in the RNAi field in the future? After two decades of scientific effort, multiple devastating clinical trial setbacks, and billions of dollars spent on research and development, RNAi therapies are finally establishing themselves in […]

July 22, 2022 - 7 minutesmins - By Farhan Mitha

News and Trends 21 Jul 2022

Almirall launches new call to find innovative therapies for skin diseases

A new call for new therapies to treat skin diseases has been launched by global Spanish-based biopharmaceutical company, Almirall, S.A. This is the seventh call through AlmirallShare – an open innovation platform for proposals to establish collaborations in dermatological research. The aim of this year’s edition is to find new partnership opportunities for assets in […]

July 21, 2022 - 3 minutesmins - By Liza Laws

News and Trends 22 Mar 2022

Biotechs Carve out Niches in Cystic Fibrosis Treatment Space

Cystic fibrosis has proved resistant to treatments that provide long-lasting respite from the sticky mucus that affects the lungs and other organs. Emerging biotechs such as Kither Biotech as well as the old guard including Vertex are finding new ways to tackle the inherited disease. Cystic fibrosis is best known for causing thick mucus in […]

March 22, 2022 - 5 minutesmins - By Anita Chakraverty

Knowledge hub

Categories

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Approaches

Therapeutic areas

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

R&D

BD

World biotech leaders

Connect with decision-makers and researchers worldwide for collaborative opportunities

Company

Join Us

Subscribe to our newsletter

Advertise with us

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Rare disease

Ionis presents positive study results at International Symposium on amyloidosis

Avacta’s drug targeting soft tissue sarcoma granted Orphan Drug Designation by FDA

Sanofi’s first treatment in the U.S. approved for adults and children with ASMD

Emactuzumab designated as an orphan medicinal product for TGCT in Europe

Amgen announces top-line results from biosimilar phase 3 study

Ocelot Bio receives FDA Orphan Drug Designation for lead candidate for treatment of hepatorenal syndrome

Vaderis emerges from stealth to start HTT trial

Emtora to use $16.9M grant to develop treatment for rare genetic disease

Aeglea BioTherapeutics’ arginine 1 deficiency treatment gets EMA ok

The coming of age story of RNAi technology

Almirall launches new call to find innovative therapies for skin diseases

Biotechs Carve out Niches in Cystic Fibrosis Treatment Space

Subscribe to our newsletter to get the latest biotech news!

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Subscribe to our newsletter

Get the latest biotech news directly to your inbox!

Newsletter Signup - Footer

Subscribe to our newsletter to get the latest biotech news!