RDR Alert®

The FDA has approved ibrutinib (IMBRUVICA) plus rituximab (RITUXAN) for the treatment of adult patients with Waldenström's macroglobulinemia, a rare and incurable type of non-Hodgkin's lymphoma.

Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.

A contributing mechanism responsible for the development of idiopathic pulmonary fibrosis has been identified.

The FDA grants an orphan drug designation to a novel gene therapy candidate for the treatment of patients with hereditary angioedema.

The FSA approves lanadelumab (Takhzyro), the first monoclonal antibody approved in the United States to treat patients 12 years and older with types I and II hereditary angioedema.

Howard Levy, MBBCh, PhD, MMM, highlights phase 2/3 trial of MarzAA for the treatment of hemophilia.

A type of electrical stimulation directed at the brain and spine may help treat the symptoms of rare movement disorders like neurodegenerative ataxias.

The FDA has granted a rare pediatric disease designation to Krystal Biotech’s KB105 for the treatment of patients with TGM-1-deficient autosomal recessive congenital ichthyosis.

The FDA approves the first drug, cenegermin, for the treatment of neurotrophic keratitis, a rare disease affecting the cornea.

Investigators have discovered a mechanism that could reduce the toxic aggregation of huntingtin protein, which is responsible for neurodegeneration in patients with Huntington’s disease.

The new test should replace the water deprivation test as the new standard of care, investigators say.

A team of investigators at Cleveland Clinic discover a novel subtype of multiple sclerosis which features neuronal loss but no demyelination of the brain’s white matter.

The FDA grants RMAT designation to AT132 for the treatment of patients with X-linked Myotubular Myopathy.

A rapid genetic diagnostic tool coupled with a sustained release therapeutic is being developed for lower-grade gliomas by a team of investigators.

The FDA has approved stiripentol (Diacomit) for the treatment of seizures associated with Dravet syndrome, a rare form of epilepsy.

The FDA has approved pembrolizumab to be used in combination with pemetrexed and platinum as first-line treatment for patients with metastatic, non-squamous non-small cell lung cancer, with no EGFR or ALK genomic tumor aberrations.

The FDA has granted an orphan drug designation to Immune Pharmaceuticals, Inc.’s bertilimumab for the treatment of bullous pemphigoid.

The FDA accepts Alexion Pharmaceutical Inc.’s BLA for review for approval of ALXN1210 for the treatment of patients with paroxysmal nocturnal hemoglobinuria.

The FDA has granted an orphan drug designation to ASLAN Pharmaceuticals’ ASLAN003 for the treatment of acute myeloid leukemia.

The FDA has granted a fast track designation to gene therapy product candidate AAV-CNGB3 for the treatment of achromatopsia caused by mutations in the CNGB3 gene.

The FDA has approved Bristol-Meyers Squibb Company’s nivolumab (Opdivo) for the treatment of metastatic small cell lung cancer (SCLC) that has progressed after platinum-based chemotherapy and at least 1 other line of therapy.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

Pauline Funchain MD, explains how better understanding of genetics can help shape future treatments for rare cancers.

Investigators from the University of Adelaide, in Australia, have found that corrected and healthy airway stem cells can replace the ones that cause cystic fibrosis and combat the genetic disorder.

A peer-reviewed manuscript outlines the preclinical validation for the use of QR-110 in the treatment of Leber’s congenital amaurosis 10 (LCA10).

The FDA granted an orphan drug designation to Rafael Pharmaceuticals, Inc.’s CPI-613 for the treatment of peripheral T-Cell lymphoma (PTCL).

The FDA has approved lenvatinib capsules (Lenvima, Eisai Inc.) for first-line treatment of patients with unresectable hepatocellular carcinoma.

A new study has identified a genetic mutation that makes individuals with epidermodysplasia verruciformis more susceptible to human papillomavirus.

The FDA has granted an orphan drug designation and a rare pediatric disease designation to Wave Life Sciences Ltd.’s WVE-210201 for the treatment of Duchenne muscular dystrophy (DMD).