Rare Disease

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5 Rare Diseases That Now Have Their First FDA-Approved Treatments

By Giuliana Grossi

February 28th 2025

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, signifying progress in closing treatment gaps for rare disease.

FDA approved. | Image Credit: Pawel - stock.adobe.com

FDA Approves Mirdametinib for Neurofibromatosis Type 1 With Plexiform Neurofibromas

By Giuliana Grossi

February 12th 2025

Gene therapy, gene editing concept | image credit: vchalup - stock.adobe.com

Delandistrogene Moxeparvovec Fails to Meet Primary End Point in Phase 3 Study

By AJMC Contributor

November 15th 2024

Patients with SMA and their caregivers stand to benefit from shared decision-making processes | image credit: Intelligent Horizons - stock.adobe.com

Shared Decision-Making Is Essential Prior to SMA-Enhancing Treatments

By Karen Jacobson-Sive

November 8th 2024

SMA type is a strong predictor of some oral functioning in affected patients | image credit: RTimages - stock.adobe.com

Oral Function Impairment Varies Across Adult Patients With SMA

By Karen Jacobson-Sive

October 28th 2024

More News

October 13th 2017

Diagnosing and Treating Neutropenia: Q&A With Dr Daniel J. DeAngelo

Before treating certain patients, Daniel J. DeAngelo, MD, JD, of Harvard Medical School and the Dana-Farber Cancer Institute, will council them on the risk of developing neutropenia, which can result in hospitalization or even an intensive care stay.

September 28th 2017

Researchers Categorize Incurable Childhood Brain Tumors as Separate Cancer Types

Christina Mattina

After examining over 1000 cases of high-grade gliomas in children and young adults, researchers have split these rare brain tumors into at least 10 different subtypes, which could help clinicians choose and deliver more effective treatments.

September 13th 2017

FDA Announces Progress, Next Steps in Streamlining Orphan Drug Review Process

Christina Mattina

FDA Commissioner Scott Gottlieb, MD, announced the agency’s progress in streamlining the orphan drug review process and outlined plans to close a loophole currently hindering pediatric drug research.

September 7th 2017

Following Pembrolizumab, Nivolumab Trials in Multiple Myeloma Paused

Surabhi Dangi-Garimella, PhD

Bristol-Myers Squibb announced that the FDA has placed a clinical hold on 3 combination trials evaluating its programmed death-1 inhibitor nivolumab (Opdivo) in patients with relapsed/refractory multiple myeloma.

September 4th 2017

Healthy, but Still Hurting: Challenges of Cancer Survivorship

Laura Joszt, MA

The assumption in the United States is that once someone with cancer is cured, done with treatment, and healthy, that they are now okay. However, the reality is that survivors contend with lingering challenges that aren’t visible and make it difficult for them to ask for help or admit they need it.

September 1st 2017

5 Significant Developments With CAR-T Therapy

Surabhi Dangi-Garimella, PhD

A look at some of the major developments in the chimeric antigen receptor T (CAR-T) cell space.

September 1st 2017

What We're Reading: HHS Cuts ACA Ads; Chemical Hazard in Houston; CAR-T Treatment Price Tag

August 31st 2017

Abeona Receives Breakthrough Designation for Rare Skin Disease Gene Therapy

A gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB), a rare skin disease that causes the skin to be very fragile and blister easily, has been granted Breakthrough Therapy designation status by the FDA.

August 30th 2017

FDA Approves Tisagenlecleucel, the First CAR-T Cell Therapy in the United States

Laura Joszt, MA

The FDA has approved the first chimeric antigen receptor-T (CAR-T) treatment, tisagenlecleucel (Kymriah), for the treatment of B-cell precursor acute lymphoblastic leukemia in certain pediatric and young adult patients. The therapy represents a new frontier in cancer care.

August 25th 2017

Case Study Identifies New Trigger for CAR-T Cell Expansion in DLBCL in JCAR017 Trial

Surabhi Dangi-Garimella, PhD

Chimeric antigen receptor (CAR)-T cells can re-expand in a lymphoma patient months after the initial infusion and can also be active against the patient’s cancer, according to a new study.

August 21st 2017

FDA Approves Besponsa for Relapsed/Refractory Acute Lymphoblastic Leukemia

Alison Rodriguez

The FDA has approved Besponsa (inotuzumab ozogamicin) for treating adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.

August 18th 2017

What We're Reading: Mylan Settlement; MS Pricing Probe; Iowa Insurer's Premium Rate Hike

August 18th 2017

Understanding Patient Preferences Can Promote Adherence in MS Patients, Study Says

Alison Rodriguez

Patient preferences for multiple sclerosis therapies are important to take into account and are increasingly so as alternatives to traditional injection therapies are developed to address adherence issues.

August 10th 2017

ICER Seeks Public Comment on Scoping Document for CAR-T Treatments

Surabhi Dangi-Garimella, PhD

The Institute for Clinical and Economic Review (ICER) has released a draft version of their scoping document that will compare the clinical effectiveness and value of 2 chimeric antigen receptor (CAR)-T cell treatments being reviewed by the FDA.

August 10th 2017

Vaccine for Glioblastoma Awarded Orphan Drug Status by FDA

Christina Mattina

MimiVax announced that its immunotherapy vaccine SurVaxM, which is being tested in clinical trials for glioblastoma, has been designated an orphan drug by the FDA.

August 9th 2017

Results of Phase 2 Study Investigating Luspatercept in MDS

Alison Rodriguez

The results of a phase 2 study that explored the effects of luspatercept in patients with lower-risk myelodysplastic syndromes (MDS) were presented at the 22nd Congress of the European Hematology Association by Acceleron Pharma Inc. The company is developing the drug with Celgene.

August 7th 2017

NCI Researchers Discuss Prospects and Challenges of Treating Glioblastoma

Christina Mattina

Amidst news of Senator John McCain’s glioblastoma diagnosis, a recent discussion between 2 neuro-oncology researchers explored the reasons why this aggressive brain tumor is so difficult to manage, and outlined the ongoing investigations into possible therapies.

August 6th 2017

Study Examines Inpatient Rehabilitation for Patients With Marfan Syndrome

Alison Rodriguez

Patients with congenital heart defects, such as Marfan syndrome (MFS), are experiencing longer life expectancies due to new advances in medicine-resulting in a greater need for medical rehabilitation.

August 5th 2017

Phase 1 and Phase 2 Study Results Find Potential Treatment for Severe Cystic Fibrosis

Alison Rodriguez

Vertex Pharmaceuticals recently announced positive data from phase 1 and 2 studies of 3 triple combination regimens in cystic fibrosis patients with one F508del mutation and one minimal function mutation (F508del/Min). These results are the first to present the potential of treating the underlying cause of those with difficult-to-treat cystic fibrosis.

August 4th 2017

5 Developments With Cancer Immunotherapy

Surabhi Dangi-Garimella, PhD

There’s a lot happening in clinical practice with immunotherapy treatments: expanded indications for nivolumab and ipilimumab, biomarkers to judge patient response to combinations, and developments with CAR-T.

This Week in Managed Care: August 4, 2017

August 4th 2017

This Week in Managed Care: August 4, 2017

This week, the top managed care stories included bipartisan proposals for fixing the Affordable Care Act (ACA); FDA approved a targeted therapy for patients with acute myeloid leukemia who have a rare genetic mutation; and a study found that more patients have gained access to clinical trials after the ACA was implemented.

August 2nd 2017

ICER Seeks Comments for Proposed Changes for Orphan Drugs Assessments

Alison Rodriguez

The Institute for Clinical and Economic Review is seeking public comments on its recently released outline of proposed adaptations involving the assessment of certain treatments for ultra-rare conditions, also known as orphan drugs.

August 2nd 2017

CAR-T Cells Can Increase Clinical Remission Rates in Multiple Myeloma Patients

Alison Rodriguez

The new immunotherapy of chimeric antigen receptor (CAR)-T cells has demonstrated the ability to increase clinical remission in multiple myeloma patients by targeting the B-cell maturation protein that participates in disease progression.

August 1st 2017

Enasidenib Approved for IDH2-Mutated Patients With Relapsed/Refractory AML

Surabhi Dangi-Garimella, PhD

Enasidenib (Idhifa), developed by Celgene and Agios, has been granted FDA approval in tandem with a companion diagnostic test to detect IDH-2 mutations.

July 30th 2017

FDA Expands Approval of Yervoy for Metastatic Melanoma in Pediatric Patients

Alison Rodriguez

The FDA has expanded the indication of Bristol-Myers Squibb’s Yervoy (ipilimumab) to include the treatment of pediatric patients 12 years of age and older with unresectable or metastatic melanoma.

July 28th 2017

Breakthrough Therapy Designation for Venetoclax in AML

Venetoclax (Venclexta), in combination with low dose cytarabine, has been granted a breakthrough therapy designation for use in elderly, treatment-naive patients with acute myeloid leukemia (AML) who cannot handle intensive chemotherapy.

July 26th 2017

Patient Questionnaires Can Predict Clinical Outcomes in Patients With Multiple Sclerosis

Alison Rodriguez

Patient-reported outcomes (PROs) can demonstrate the effects of a disease or treatment. By using patient questionnaires, the results can be analyzed to predict clinical outcomes in medical research for diseases, including multiple sclerosis (MS).

July 25th 2017

Mechanism for Checkpoint Inhibitor Resistance Identified in Invasive Bladder Cancer

Christina Mattina

Researchers have identified a cellular signaling pathway that when activated by mutations may prevent immune cells from infiltrating bladder tumors, thus making the cancer resistant to immune checkpoint inhibitors.

July 25th 2017

What We're Reading: Monthly HIV Shot; Molina Layoffs; Charlie Gard Treatment

July 20th 2017

Rare Diseases Create Significant Care and Financial Burdens Among Patients

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