Therapeutic-Specific Areas

The agency has approved Stemline Therapeutics’ Elzonris (tagraxofusp-erzs) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm for both adults and pediatric patients.

Agilent and other partners are funding development of Tapestri, a single-cell sequencing platform designed to help predict cancer relapse in individual patients and show the efficacy of gene-editing experiments.

FDA has approved Truxima (rituximab-abbs), a biosimilar to Roche’s anti-cancer biologic, Rituxan (rituximab).

The companies aim to advance research into inflammatory bowel disease.

The acquisition will give Genentech full rights to Jecure’s preclinical portfolio of NLRP3 inhibitors.

Charles River Laboratories has announced a data expansion in its compendium of tumor models for oncology drug R&D.

The agency has approved two new chemical entities, Daurismo (glasdegib) from Pfizer, and Vitrakvi (larotrectinib) from Loxo Oncology, for treating cancers.

The companies will develop AMG 714, a novel anti-IL-15 monoclonal antibody, for the treatment of gluten-free diet non-responsive celiac disease in a collaboration worth $170 million.

The companies will develop treatments for a range of neurological and inflammatory diseases.

While recognizing the unsung work of scientists in corporate research and development, the Galien Awards remind the industry of its priorities: patients and future patients within the global community.

More-and earlier-interaction between R&D and payers will be essential if innovative therapies are to become more accessible for patients, and more profitable for manufacturers, said panelists at the 2018 Galien Foundation Forum.

AbbVie will assume full development and commercial responsibility for its collaboration with Galapagos to discover and develop new therapies to treat cystic fibrosis (CF).

The companies will work together to advance a number of Morphic's oral integrin therapeutics for fibrosis-related indications in a deal with $100 million.

The companies will work together to develop Ionis-FB-LRx for the treatment of complement-mediated diseases, including Geographic Atrophy (GA), the advanced stage of dry age-related macular degeneration (AMD), in a deal worth $760 million.

Novartis plans to acquire Endocyte, a biopharmaceutical company, to accelerate the development of innovative radioligand technology for treating cancer.

The new guidance, Rare Diseases: 2 Early Drug Development and the Role of Pre-IND Meetings Guidance for Industry, discusses the planning of pre-IND meetings with FDA and sponsors.

Roche has acquired Tusk Therapeutics in a deal worth up to EUR 659 million (US$762 million).

The companies will develop and commercialize ARO-HBV, a Phase I/II subcutaneous, ribonucleic acid interference therapy candidate being investigated for the treatment of chronic hepatitis B viral infection.

The companies will work together to discover, develop, and commercialize immunotherapies for patients with solid-tumor cancers in a collaboration worth $695 million per program.

The National Institute for Health and Care Excellence recently rejected National Health Service’s funding of Kymriah for diffuse large B-cell lymphoma (DLBCL) despite recognizing that the drug has significant clinical benefits.

The European Medicines Agency has recommended Luxturna (voretigene neparvovec) as the first treatment option for hereditary retinal dystrophy with mutations of the RPE65 gene.

The companies will develop therapies targeting the in-vivo elimination of hepatitis B virus (HBV) with Precision’s proprietary genome editing platform.

The agency approved AstraZeneca’s Lumoxiti (moxetumomab pasudotox-tdfk) injection for intravenous use for the treatment of adult patients with relapsed or refractory hairy cell leukemia.

The pharmaceutical, clinical, and bioanalytical contract solutions provider has implemented advanced techniques for the collection and use of peripheral blood mononuclear cells (PBMCs) for early-phase clinical trials at its Clinical Pharmacology Unit in Antwerp, Belgium.

Increasingly complex trial protocols have added to IMP manufacturing challenges.

The European Commission (EC) has approved Novartis’ chimeric antigen receptor T cell (CAR-T) cell therapy Kymriah for the treatment of B-cell acute lymphoblastic leukemia and relapsed or refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy.

Dompé Farmaceutici’s Oxervate (cenegermin), a topical eye drop, is the first treatment approved by the agency for neurotrophic keratitis, a rare disease affecting the cornea.

The acquisition will strengthen Astellas Pharma’s position in ophthalmology.

Researchers at the University of Pennsylvania found that diabetes drugs called thiazolidinediones can promote the metabolism of glutamine to help control disease-causing inflammation.

Stem-cell developer TiGenix has been acquired by Takeda Pharmaceutical for approximately EUR 520 million (US$604 million).