Rare Diseases

FDA approves $4.25 million gene therapy for MLD

March 29, 2024 - Once again, the pharmaceutical industry has topped its existing astronomical prices with Lenmeldy, an FDA-approved gene therapy for children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD), which has a wholesale acquisition price tag of $4.25...

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FDA Launches New Advisory Committee for Genetic Metabolic Diseases

by Veronica Salib

Adding to the lengthy list of advisory committees under its belt, the United States Food and Drug Administration (FDA) announced its plans to create a new advisory committee to specialize in genetic...

Melanoma Drugs Repurposed Can Shrink Rare Brain Tumors

by Veronica Salib

A study published last week in the New England Journal of Medicine concluded that BRAF–MEK inhibitors — traditionally used in melanoma treatment — can shrink papillary...

FTC’s Lawsuit Against Amgen Horizon Deal to Reach Court in September

by Hayden Schmidt

Federal Trade Commission (FTC) officials are taking Amgen and Horizon to court this September to block a prospective pharmaceutical acquisition of the two companies. The FTC announced that it planned...

FNIH Enhances Rare Disease Research by Adding 8 Diseases to BGTC

by Veronica Salib

On May 16, 2023, the Foundation for the National Institutes of Health (FNIH) announced eight rare diseases to be explored by the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC). As one of the newest additions...

Drug Repurposing May Benefit Rare Disease, Considerations, and Caveats

by Veronica Salib

The Orphan Drug Act of 1983 defines a rare disease as a medical condition that impacts less than 200,000 people in the United States. Despite the restrictive nature of this definition, the Genetic and Rare Diseases Information Center of...

Amgen’s $27.8 Billion Rare Disease Drug Acquisition

by Hayden Schmidt

On Monday, Amgen Inc announced its acquisition of the rare disease pharmaceutical company Horizon Therapeutics PLC, building on its portfolio of key drugs for treating uncommon conditions. Amgen...

FDA Approves $3.5 Million per Dose Hemophilia B Gene Therapy

by Hayden Schmidt

Last week, the FDA’s Center for Biologics Evaluation and Research announced the approval of Hemgenix, an adeno-associated virus vector-based gene therapy for adults with hemophilia B (congenital...

Understanding the Complexities Associated with IgA Nephropathy

by Veronica Salib

According to the Mayo Clinic, IgA nephropathy — sometimes referred to as Berger’s disease — is an autoimmune disease in which an antibody called immunoglobulin A (IgA) builds up in the kidneys, leading to local...

FDA and NIH Launch Partnership for Rare Neurodegenerative Diseases

by Veronica Salib

Earlier this week, the FDA and NIH announced their launch of a public–private partnership for research on rare neurodegenerative diseases. The partnership, called the Critical Path for Rare...

FDA Approves Cell-Based Gene Therapy for Patients with Beta Thalassemia

by Veronica Salib

On August 17, 2022, the FDA approved bluebird bio's Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy to treat patients with beta thalassemia who need regular red blood...

Pfizer Buys Global Blood Therapeutics for $5.4B, Continues Acquisition Splurge

by Hayden Schmidt

Pfizer is back at it with another acquisition, this time scooping up the rare disease pharmaceutical company Global Blood Therapeutics for $5.4 billion in cash. Pfizer’s acquisition will cede...

Bluebird Bio’s Rare Disease Gene Therapies Recommended for FDA Approval

by Hayden Schmidt

Last week, the FDA Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 15 – 0, concluding that the benefits of bluebird bio’s gene therapy to treat cerebral...

FDA Refuses to File Aeglea Biologics License Application

by Hayden Schmidt

Aeglea BioTherapuetics announced that it had failed to secure a biologics license application (BLA) from the FDA after administrators issued a refusal to file for the company’s biologic...

Novartis Gains FDA Approval for Follicular Lymphoma Cell Therapy

by Hayden Schmidt

Novartis’s Kymriah was granted accelerated FDA approval last week after the drug’s clinical trial results demonstrated an 86% overall response rate and a high level of safety. Patients with...

Novo Nordisk, Flagship to Develop Rare Disease Treatments

by Samantha McGrail

Novo Nordisk and Flagship Pioneering recently collaborated to create a portfolio of novel research programs to develop transformation metabolic and rare disease treatments. The companies will apply...

AstraZeneca Launches R&D Challenge to Advance Drug Discovery

by Samantha McGrail

AstraZeneca recently launched a global research and development Postdoctoral Challenge to help advance drug discovery for some of the world’s most complex diseases.  The challenge invites...

AstraZeneca, Alexion, Neurimmune to Develop Rare Disease Antibody

by Samantha McGrail

AstraZeneca Rare Disease and Alexion group recently closed a global collaboration and license agreement with Neurimmune AG for the investigational human monoclonal antibody, NI006. NI006 is a...

Pfizer, Beam Focus on Precision Medicine for Rare Diseases

by Samantha McGrail

Pfizer and Beam Therapeutics recently entered into a four-year collaboration focused on in vivo base editing programs to generate precision medicine for rare diseases of the liver, muscle, and central...

AstraZeneca Enters Agreement for Transthyretin Amyloidosis Medicine

by Samantha McGrail

 AstraZeneca recently entered into a global development and commercialization agreement with Ionis Pharmaceuticals for transthyretin amyloidosis medication, eplontersen. Eplontersen is a...

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