RDR Alert®

The FDA has approved amifampridine (Firdapse) tablets as the first ever treatment of the rare autoimmune disorder, Lambert-Eaton myasthenic syndrome (LEMS) in adults.

The FDA has approved gilteritinib (XOSPATA) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test.

The FDA has approved rituximab-abbs (Truxima) for the treatment of adult patients with non-Hodgkin’s lymphoma. This indication stands as the first FDA-approved biosimilar for the rare blood cancer.

Investigators in Denmark found that both current and ex-smokers have an increased risk of developing myeloproliferative neoplasms.

Mayo Clinic investigators found young patients with myeloproliferative neoplasms who are under age 40 are a steadily growing population and seem to have longer survival than their older counterparts.

The FDA has granted an accelerated approval to larotrectinib (Vitrakvi, Bayer and Loxo Oncology, Inc) for the treatment of pediatric and adult solid tumors with neurotrophic receptor tyrosine kinase (NTRK) gene fusion.

The recently approved phenylketonuria (PKU) drug, pegvaliase, is helping patients experience less disease burden—and drastically in some cases.

The drug is approved as a treatment for adult patients with newly-diagnosed acute myeloid leukemia (AML) who are 75 years or older, or who have comorbidities that preclude the use of intensive induction chemotherapy.

Emapalumab-lzsg was approved by the FDA for the treatment of primary hemophagocytic lymphohistiocytosis in pediatric and adult patients with refractory, recurrent, or progressive disease, or intolerance to conventional therapy.

The FDA has expanded Novartis’ eltrombopag (Promacta) to include first-line treatment for adults and pediatric patients aged 2 years and older with severe aplastic anemia (SAA) in combination with standard immunosuppressive therapy (IST).

Here’s a round-up of recent designations granted by the FDA to products developed to treat rare diseases.

New research contradicts previous studies supporting the use of inositol supplementation in premature infants at risk for an eye condition that could lead to blindness.

Brentuximab vedotin (ADCETRIS, Seattle Genetics) is approved in combination with chemotherapy for adults with previously untreated systemic anaplastic large cell lymphoma (sALCL) or other CD30-expressing peripheral T-cell lymphomas (PTCL), including angioimmunoblastic T-cell lymphoma and PTCL not otherwise specified.

The investigators hope to gain FDA approval on the treatment so they can treat more patients.

A new, fundamental biological process in CLN8 disease, a form of Batten disease, has been discovered by a team of investigators from Baylor College of Medicine.

Treatment with crizanlizumab reduced pain in patients with sickle cell disease who were experiencing vaso-occlusive crisis.

Aquestive Therapeutics’ clobazam (SYMPAZAN) oral film has been approved by the US Food and Drug administration (FDA) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in patients aged 2 years or older.

The use of marijuana for patients with multiple sclerosis can have a limited effect on symptoms such as spasticity, pain, and bladder dysfunction.

The chip was developed using cells from patients with and without ALS.

Robert Chen, MD, and Hodgkin’s lymphoma patient, Jeremy Hernandez, speak on the challenges of receiving and treating a rare cancer diagnosis in young adults.

The brain’s lymphatic vessels may offer a new way to treat multiple sclerosis.

When successful, these relationships provide valuable insights to support strategies in many areas, including planning for clinical research, patient recruitment and education, reimbursement, access, compliance, and other areas.

Treating the blood clotting disorder thrombotic thrombocytopenic purpura might be possible through the use of a therapeutic enzyme.

Here’s a round-up of recent designations granted by the FDA to products developed to treat rare diseases.

Men with Fabry disease develop white matter lesions much quicker than their female counterparts.

A team of investigators has found that collaborative efforts among several leading medical institutions have helped increase the study of new medications for myelodysplastic syndromes (MDS).

Interferon‐α2 and ruxolitinib combination therapy shows promise in treating myeloproliferative neoplasms, according to recent preliminary findings.

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

From August 2014 to September 2018, a total of 386 confirmed cases of acute flaccid myelitis across the United States has been confirmed by the CDC.

Since AHC is a rare neurological disease, finding a diagnosis can be a harrowing experience.