Asthma

Atrial Fibrillation

Autoimmune Diseases

Biologics

Biosimilars

Blood Cancer

Breast Cancer

COVID-19

Cardiovascular Diseases

Depression

Dermatology

Diabetes

Digital Health

Drug Coverage

Eyecare

Friedreich Ataxia

Gene Therapy

Graft Versus Host Disease

Heart Failure

Hemophilia

Hypertrophic Cardiomyopathy (HCM)

Leukemia and Lymphoma

Liver Disease

Lung Cancer

Mental Health

Multiple Sclerosis

NSCLC

Neonatal Care

Obstetrics-Gynecology & Women's Health

Oncology

Opioids

PTSD

Pain

Pediatrics

Plaque Psoriasis

Population Health

Post-Acute Care

Prescription Digital Therapeutics

Pulmonary Arterial Hypertension

Reproductive Health

Respiratory Conditions

Safety & Recalls

Skin Cancer

Sleep Disorders

Solid Tumors

Spinal Muscular Atrophy

Substance Use Disorder

The Improving Patient Access Podcast

Type I Diabetes

Type II Inflammation

Ulcerative Colitis

Urology

Urothelial Carcinoma

Vaccines

Vaccines: 2023 Year in Review

Women's Health

News

Media

Between the Lines

Expert Interviews

K-Cast

Medical World News

Podcasts

Population Health Perspectives

Conferences

Partners

Publications

All Publications

MHE Publication

Formulary Watch Supplements

Formulary Watch Spotlights

Supplements And Featured Publications

Events

2024 PBMI Annual National Conference

PBMI MAP

Virtual Events

CME/CE

Resources

Emerging Leaders

Interactive Tools

PBM Leaders

Subscribe

Articles

Poor balance and coordination are prominent features of Friedreich’s ataxia (FRDA). Czech researchers found that neuropsychiatric symptoms are also common among patients with FRDA. 

Researchers to Clinicians: Don’t Neglect the Neuropsychiatric Symptoms of Friedreich’s Ataxia

Vutrisiran is the generic form of previously approved Amvuttra. The FDA’s target date for the treatment of transthyretin amyloidosis with cardiomyopathy is March 23, 2025.

FDA Accepts sNDA ATTR Amyloidosis Drug, Vutrisiran

Imkeldi is a new formulation of imatinib approved as a strawberry-flavored, shelf-stable liquid designed to be more appealing to a wider range of patients, pediatric patients included. 

FDA Approves New Imatinib Oral Solution

Greg Baker, CEO of AffirmedRx, shared with MHE editors how the company is addressing ongoing challenges in the pharmacy benefit management (PBM) industry, particularly corporate consolidation and transparency issues. 

AffirmedRx’s Efforts to Address PBM Transparency and Drive Industry Change

Managed Healthcare Executive provides C-suite executives at health plans and provider organizations with news, analysis, insights, and strategies to pursue value-driven solutions.

About Us

Advertise

Editorial

Contact Us

Terms and Conditions

Privacy

Do Not Sell My Personal Information

Managed Healthcare Executive

Facebook

LinkedIn

Resource Centers

Eye Care

Spotlight

ID Week 2024

AMCP Nexus 2024

AHIP

AIDS Conference

ATS 

Business

Business Strategy

Chronic Disease

Consumers

Drug Costs

Drug Pipeline

Formulary

Health Management

Health/Disease Strategy

Hospitals & Providers

Industry Analysis

Pharmacy

Politics and Policy

Practice Management

Reimbursement

Technology

1rem

Authors

Celltrion USA is Committed to Delivering Advancements in IBD Treatments

A new study shows that gene therapy may benefit spinal muscular atrophy (SMA) patients who require breathing support through tracheostomy — a group typically excluded from clinical trials.

, examined data from the RESTORE registry for 34 patients with tracheostomies who received Zolgensma (onasemnogene abeparvovec). Led by Laurent Servais, M.D., of the University of Oxford, the study represents the first significant analysis of gene therapy outcomes in this high-risk population.

Thirty-three of the patients had two SMN2 gene copies, while one patient had a single copy. Most patients (88%) were diagnosed clinically rather than through newborn screening. The median age at diagnosis was 3.5 months, with patients receiving initial treatment at 5 months and requiring tracheostomy at approximately 9 months.

"There were no new safety findings with [Zolgensma] treatment in patients with SMA with tracheostomy across real-world settings," the study authors wrote. While four patients experienced serious treatment-related adverse events, these aligned with Zolgensma's known safety profile. Two deaths occurred during the study period, neither related to the treatment.

The findings suggest that patients requiring tracheostomy — who are often considered too ill for gene therapy — may still benefit from treatment. Eight patients received Zolgensma alone, while 26 received additional therapies before or after gene therapy.

Without treatment, nearly all patients with type 1 SMA, the most severe form, require respiratory assistance within their first year of life. Historically, ventilated SMA patients had a life expectancy of just two to three years. However, early diagnosis through newborn screening programs, combined with timely treatment initiation, has shown promise in delaying or reducing the need for ventilatory support.

 has shown that Infants with spinal muscular atrophy (SMA) who receive a gene therapy in the first year of life can typically avoid permanent ventilation. A different 

 published in August showed that patients with SMA who were on long-term ventilation via tracheostomy before receiving Zolgensma showed significant improvements in respiratory function over a mean follow-up period of 7.6 months.

Both of these studies add to the body of evidence showing potential benefits and improvements for older SMA patients receiving gene therapy.

The study drew on data from the RESTORE registry for 34 patients with tracheostomies who received Zolgensma (onasemnogene abeparvovec). 

Clinical

Gene Therapy Shows Promise for SMA Patients with Tracheostomies

The FDA has approved a new Imatinib oral formulation called Imkeldi to treat specific leukemias and other cancers in pediatric patients over the age of one and adults, according to a news release published 

. It was developed by Shorla Oncology and approved for 10 indications. Imatinib was first approved as a tablet with the name Gleevec in 2001 and is marketed by Novartis.

“Oral solutions may ensure more precise and consistent dosing, offering a convenient alternative to compounding for patients who have difficulty swallowing or require dosing tailored to body surface area,” Sharon Cunningham, CEO of Shorla said in the news release.

Imkeldi is a tyrosine kinase inhibitor, which means it can slow or prevent the growth of cancers, including chronic myeloid leukemia (CML), acute lymphoblastic leukemia, myelodysplastic syndrome /myeloproliferative disease (MDS/MPD) and gastrointestinal tumors (GIST).

This year, an estimated 9,280 people will be diagnosed with CML, over 10,000 with MDS/MPD and up to 6,000 with GIST, the press release says.

Specific indications include but are not limited to:

Newly diagnosed adult and pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase.

Pediatric patients with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy.

Treatment of adult patients with relapsed or refractory Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL).

Adult patients with myelodysplastic/myeloproliferative diseases (MDS/MPD) associated with platelet-derived growth factor receptor (PDGFR) gene re-arrangements.

Patients with Kit (CD117) positive unresectable and/or metastatic malignant gastrointestinal stromal tumors (GIST).

Dosage is dependent on indication. Doses of 400 mg and 600 mg should be given once a day and an 800 mg dose should be administered as 400 mg twice a day. All doses should be taken with a large glass of water and a full meal.

The most common adverse reactions reported were edema, nausea and vomiting, according to the 

Superficial edema was also seen in 2-6% of CML patients treated with Imatinib. In GIST patients, severe fluid retention was seen in 9 - 13.1%.

Liver function should also be monitored, as well as signs of hematologic toxicity. Complete blood counts should be performed weekly for the first month of treatment and biweekly for the second month.

Grapefruit juice should be avoided during treatment with Imatinib. Breastfeeding women should not take Imatinib.

Imkeldi will arrive in the United States on Jan. 6, 2025 and customer shipments will begin no later than Jan. 13, Rayna Herman, CCO of Shorla said in an email. It will cost $2,350 per bottle. 

Formulary Watch

The FDA has approved BridgeBio’s Attruby (acoramidis), an orally administered therapy to treat adults with transthyretin amyloid cardiomyopathy (ATTR-CM) to reduce cardiovascular death and cardiovascular-related hospitalization.

ATTR-CM is a type of heart failure. It is a rare disease that leads to a build-up of the protein transthyretin in the left ventricle of the heart, leading to a thickening of the heart muscle.

“Access to this new therapy means more hope and more opportunity to improve the lives of patients with amyloidosis,” Muriel Finkel, president of Amyloidosis Support Groups, a non-profit organization dedicated to the support of amyloidosis patients and caregivers, said in a 

Attruby is an oral small molecule stabilizer of transthyretin. It was designed to mimic the protective effects of T119M mutation, which has been associated with reduced risk of vascular events. It will have a list price of $18,759 for a month’s supply.

BridgeBio offers a patient support services program, ForgingBridges, which includes insurance resources and financial assistance options. Commercial patients may be eligible for a $0 copay; Medicare patients will have a max copay of $167 per months, according to company officials.

The FDA approval is based on positive results seen in the ATTRibute-CM phase 3 study, which enrolled 632 patients with symptomatic ATTR-CM, and patient were randomly assigned to receive either Attruby or placebo. The trial met its primary endpoint of a four-component composite endpoint of ACM, including CVH, N-terminal prohormone of brain natriuretic peptide (NT-proBNP), and six-minute walk distance with a Win Ratio of 1.8. The 

 is a method for reporting composite endpoints that gives priority to certain events. Patients in these trials are matched based on risk profiles and events are compared among the pairs and assigned a “win” if they meet the endpoint.

The study found that Attruby demonstrated a statistically significant treatment effect at 30 months on the Kansas City Cardiomyopathy Questionnaire and 6-minute walk test. Additionally, the increase in NT-proBNP on treatment was about half that of placebo.

BridgeBio also recently released data from an open-label extension acoramidis in ATTR-CM at the American Heart Association (AHA) Scientific Sessions and 

The open-label extension includes 389 participants who completed the 30-month. ATTRibute-CM phase 3 study confirmed results from the earlier study and found reduction of composite all-cause mortality and cardiovascular-related hospitalizations by 46% at month 36 and 48% at month 42. The open label study also found evidence of early benefit in patients who crossed over from placebo to acoramidis after month 30, compared with extrapolated placebo curve.

Attruby (acoramidis) is a small molecule approved to treat adults with transthyretin amyloid cardiomyopathy. It will have a list price of $18,759 for a month’s supply.

FDA Approves Attruby for Heart Failure Indication

Institute for Clinical and Economic Review (ICER)

 has announced plans to assess the clinical efficacy and value of tolebrutinib in the treatment of secondary progressive multiple sclerosis (SPMS). Public discussion and review panel voting is planned for June 2025.

Tolebrutinib is an investigational Bruton’s tyrosine kinase (BTK) inhibitor being developed by Paris-based Sanofi for the treatment of relapsing forms of MS as well as nonrelapsing SPMS (nrSPMS). Currently, there is no approved treatment for this form of MS. The oral brain penetrant performed well in the phase 3 HERCULES trial, which met the primary end point of delaying time to onset of confirmed disability progression in patients with nrSPMS. Sanofi plans to file for FDA approval by the end of the year.

ICER will assess the health and economic outcomes of tolebrutinib for nrSPMS compared to supportive care, including pharmacological and nonpharmacological treatments targeting MS symptoms not treated with disease-modifying treatments. These can include pain, numbness, fatigue, urinary incontinence and issues with cognitive function.

Objectives will include patient-related outcomes, such as disability progression, quality of life, cognitive function, depression and adverse events. Another outcome of interest is liver toxicity, with has plagued clinical trials of several BTK inhibitors, including tolebrutinib. MRI activity and disease impact on caregivers will also be assessed.

Evidence for treatment effectiveness will be derived from randomized trials, systematic reviews, and cohort studies. ICER will also consider input from patients and patient advocates, information gathered from regulatory documents, and other input from manufacturers.

The report will also include a lifetime cost-effectiveness analysis of tolebrutinib versus best supportive care in adults with nrSPMS. The analysis will include direct medical costs, such as drug administration and monitoring, and indirect costs, such as factors affecting patient and caregiver productivity.

Lastly, the review will include an assessment of benefits beyond health and special ethical priorities. These factors include whether there is a substantial unmet treatment need, the condition is of significant relevance to racial or ethnic groups that have been affected by disparities in healthcare systems, the treatment is likely to improve the caregiver’s quality of life, or the treatment’s mechanism of action or delivery method has the potential to improve treatment access.

The investigational Bruton’s tyrosine kinase performed well in the phase 3 HERCULES trial. 

ICER to Assess Sanofi’s Tolebrutinib for Secondary Progressive MS

In a recent conversation with Greg Baker, CEO of AffirmedRx, the PBM expert outlined the company’s distinct approach in the PBM industry with MHE editors.

Baker, a member of Managed Healthcare Executive's Editorial Advisory Board, expressed that AffirmedRx prioritizes the public good over profits, aiming to eliminate conflicts of interest and enhance transparency.

Greg Baker PBMS AffirmedRx

The company charges a flat per-prescription administrative fee, which ensures financial decisions are patient-focused rather than driven by drug costs. AffirmedRx also provides clients with full visibility into claims and manufacturer revenues, fostering trust and accountability.

“We're to the point where we could credential our clients directly into the adjudication system... allow them to see all the claims, what's getting paid, then they know exactly what they should be paying,” he said. “Everything is in one place, so it makes it very transparent.”

By aligning its goals with clients and patients, Baker shared that AffirmedRx differentiates itself from traditional PBMs and those backed by private equity.

For example, regulatory changes, such as those under the Consolidated Appropriations Act, reinforce the company’s mission of ethical and transparent practices, setting a standard for the evolving PBM landscape.

In a recent conversation with Greg Baker, CEO of AffirmedRx, the PBM professional outlined the company’s distinct approach in the PBM industry with MHE editors.

A Transparent, Patient-First Approach Amid Ongoing PBM Industry Challenges

Like many novel therapies, gene therapies are not without challenges that can be solved through a combination of policy and market changes. In order to improve patient access to these life-saving therapies new payment solutions, education efforts around the availability of these treatments and their benefits and risks, and the use of appropriate value assessment methods are all needed, according to a white paper from the National Pharmaceutical Council (NPC).

Journal of Comparative Effectiveness Research

There are a total of 14 gene therapies are now available for 

 and some cancers, and these potentially curative therapies often only require a single treatment, which can be complicated to deliver. The finances of paying for costly gene therapies is a challenge in the U.S., as well. Lenmeldy (atidarsagene autotemcel) is the 

, with a price tag of $4.25 million. The therapy is approved to treat metachromatic leukodystrophy, a rare disorder that damages the nervous system. The 

 starts around 2 years of age or younger and is the most common form. It progresses fast and the mortality rate is high.

“The unique characteristics of gene therapy treatments, in conjunction with uncertainties about long-term durability and future market competition, necessitate the development of innovative models to assess the value of these treatments, promote appropriate payment and advance meaningful patient access while maintaining incentives for future research and development,” the authors wrote.

In addition to the high cost of these therapies, they are faced with challenges related to production and distribution, as well as the logistical and treatment administration obstacles that can create barriers to accessing gene therapies. A growing number of gene therapies require extracting cells from the patient, isolating and genetically modifying the cells and then re-administering them to the patient. This is a complex and time-intensive processes, which limits the ability to scale production of these therapies.

For patients and caregivers, there is the added burden of special diagnostic testing and treatment administration, which can require that they travel long distances from home, sometimes necessitating staying in another location for a significant amount of time. Only specific providers and locations that are qualified for handling gene therapies can administer them.

Coverage by health plans varies greatly for gene therapies, which “holds important implications for equitable patient access to life-changing treatments,” the NPC authors noted. Medicaid’s coverage is more restrictive than the FDA approved label more than two-thirds (68.3%) of the time, and commercial plans have more restrictive policies more than half (53.5%) of the time.

Gene therapies have a lot of potential to improve health outcomes for patients with life-threatening diseases but face a number of barriers that restrict access.

Thomas Nusbickel, Chief Commercial Officer, Celltrion USA

Articles

Latest Updated Articles