Research Triangle Park, N.C.  – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).

FDA grants rare pediatric disease designation to incentivize the development of new treatments for serious and life-threatening diseases that primarily affect children aged 18 years or younger, with fewer than 200,000 people affected in the US. If AB-1003 is approved, AskBio may qualify for a priority review voucher based on receipt of this designation. A priority review voucher can be applied to another therapy in the company’s pipeline, enabling a shorter review timeline during marketing application review or can be sold and transferred to another company.

Orphan designation provides orphan status to drugs and biologics for rare diseases that meet certain criteria and potentially gives a company exclusive marketing rights for a seven-year period, along with other benefits.

“These designations for AB-1003 are clear recognition of the significant unmet medical need in LGMD, including type 2I/R9, which is the focus of AskBio’s clinical program and for which there is no approved therapy,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “The burden of this rare form of muscular dystrophy on patients and their families is profound, and these decisions support our efforts to potentially bring a new therapeutic option to people living with the 2I/R9 type of this devastating disease.”

LGMD2I/R9 is a form of LGMD caused by changes in the FKRP gene and is associated with weakness and wasting of arm and leg muscles. People living with LGMD2I/R9 may notice symptoms including loss of mobility, impaired heart or lung function. These symptoms can occur in school age and younger children. As symptoms worsen, individuals generally require wheelchairs. LGMD2I/R9 is a rare disease, estimated to affect fewer than 5,000 people in the US. Currently, there is no treatment that modifies disease progression, and management is based on the signs and symptoms present in each individual.

With a broad portfolio of investigational gene therapies at various stages of research and development, AskBio continues to develop adeno-associated virus (AAV)-based therapies to treat some of the world’s most debilitating diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications and aims to deliver breakthrough treatments that could potentially benefit tens of millions of patients worldwide.

The Food and Drug Administration granted accelerated approval to asciminib (Scemblix, Novartis AG) for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP).

The efficacy of asciminib for newly diagnosed Ph+ CML in CP was evaluated in ASC4FIRST (NCT04971226), a multicenter, randomized, active-controlled, open-label trial. A total of 405 patients were randomized (1:1) to receive either asciminib or investigator-selected tyrosine kinase inhibitors (IS-TKIs) (imatinib, nilotinib, dasatinib, or bosutinib). The main efficacy outcome measure was major molecular response (MMR) rate at 48 weeks. The MMR rate at 48 weeks was 68% (95% CI: 61, 74) in the asciminib arm and 49% (95% CI: 42, 56) in the IS-TKIs arm (difference 19% [95% CI: 10, 28], p-value <0.001). Within the imatinib stratum, the MMR rate was 69% (95% CI: 59, 78) in the asciminib arm and 40% (95% CI: 31, 50) in the IS-TKIs arm (difference 30% [95% CI: 17, 42], p-value <0.001).

In the pooled safety population in patients with newly diagnosed and previously treated Ph+ CML in CP, the most common adverse reactions (≥20%) were musculoskeletal pain, rash, fatigue, upper respiratory tract infection, headache, abdominal pain, and diarrhea. The most common laboratory abnormalities (≥40%) in patients with newly diagnosed Ph+ CML in CP were decreased lymphocyte count, decreased leukocyte count, decreased platelet count, decreased neutrophil count, and decreased calcium corrected.

The recommended asciminib dosage is 80 mg taken orally once daily at approximately the same time of day or 40 mg taken orally twice daily at approximately 12-hour intervals.

PRINCETON, N.J - Protega Pharmaceuticals Inc. recently announced that the U.S. Food and Drug Administration (FDA) has approved ROXYBOND™ (oxycodone hydrochloride) immediate-release (IR) CII 10 mg tablet for the management of pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate. ROXYBOND is the first and only FDA-approved abuse-deterrent IR 10 mg oxycodone formulation that is expected to reduce abuse by intranasal and intravenous routes.

ROXYBOND is formulated with SentryBond™ abuse-deterrent technology. This patented technology combines inactive excipients with active pharmaceutical ingredients to make the tablet more difficult to manipulate for misuse and abuse, even if it is subjected to physical manipulation and/or chemical extraction. SentryBond is designed to maintain the intended release profile of extended-release (ER) products and delay the release of IR products.

"The FDA approval of ROXYBOND 10 mg with SentryBond is a significant milestone for Protega and fulfills an unmet need for an IR opioid with abuse-deterrent technology that may reduce misuse and abuse while providing pain relief to medically appropriate patients when used as indicated," said Paul Howe, Chief Commercial Officer of Protega. "When manipulated, our innovative technology renders the pill more difficult to misuse or abuse, such as being cut or crushed to snort or inject."

Protega's SentryBond technology is a first-of-its-kind abuse-deterrent patented technology. It is designed to provide multiple levels of protection that resist physical manipulation, chemical extraction, and manipulation or transformation for injection. Protega's proprietary SentryBond technology platform could potentially be utilized in other medications to help deter misuse and abuse, e.g., hydromorphone, hydrocodone, and attention deficit hyperactivity disorder (ADHD) medications. While these uses are currently not available and require FDA approval, the technology can help in a variety of medications.

"The development of ROXYBOND with SentryBond is a step forward in fighting the national epidemic of prescription opioid overdose," said Eric Kinzler, Ph.D., VP Medical and Regulatory Affairs for Protega. "Protega is dedicated to our mission to block the path to abuse and work with healthcare professionals across the continuum of care to reduce misuse and abuse. We look forward to responsibly launching ROXYBOND 10 mg and advancing our innovative technology platform for potential application in other commonly abused prescription medications."

More than 2000 in vitro tests were conducted to demonstrate ROXYBOND tablets were difficult to manipulate vs oxycodone IR, this data, along with the results of the human abuse potential study, suggest that the physicochemical properties of ROXYBOND are expected to reduce abuse via the intranasal and intravenous routes of administration. However, abuse is still possible by intranasal, intravenous, and oral routes.

In addition to the FDA approval for the 10 mg tablet, ROXYBOND was previously approved and is already available in 5 mg, 15 mg, and 30 mg tablets. Protega plans to launch ROXYBOND 10 mg before the end of the year, providing clinicians with another risk mitigation tool they can use when treating patients with severe pain.

The addition of ROXYBOND 10 mg can enhance flexibility and precision in opioid therapy, aiming to support both physicians and patients in achieving more effective and safer pain management outcomes. For patients, the range of doses can provide better pain control, reduce the risk of side effects, and provide a smoother transition during dosing transitions. For physicians, it can allow for more flexible dosing for pain levels, better titration, and help optimize risk management across diverse patient populations.

The U.S. Centers for Disease Control and Prevention (CDC) has revised its recommendation for the pneumococcal vaccine, lowering the suggested starting age from 65 to 50. This change, announced by CDC Director Dr. Mandy Cohen, reflects the increased risk of pneumococcal infections as adults age, with a goal to enhance protection against serious pneumococcal diseases for middle-aged adults.

“Lowering the age for pneumococcal vaccination enables more adults to protect themselves against pneumococcal disease at an age when susceptibility to infection significantly rises,” Dr. Cohen stated.

Pneumococcal bacteria can lead to severe health complications such as pneumonia, meningitis, and bloodstream infections, with older adults particularly vulnerable to these diseases. The decision follows a CDC advisory panel vote of 14-1 in favor of the change, which Dr. Cohen approved shortly after.

Previously, pneumococcal vaccination was recommended primarily for two high-risk groups: children under 5 years and adults aged 65 and older. However, individuals of other ages with certain health conditions are also advised to receive the vaccine.

Streptococcus pneumoniae and related pneumococcal strains are responsible for pneumococcal disease. Each year, an estimated 150,000 hospitalizations occur due to pneumococcal pneumonia in the U.S., with the condition often complicating cases of influenza, according to CDC data. Pneumococci represent the leading bacterial cause of pneumonia in children under 5 and account for 10% to 30% of adult community-acquired pneumonia cases.

The first pneumococcal vaccine was licensed in the U.S. in 1977, and since then, four vaccine types have become available. These include Capvaxive by Merck, which protects against 21 types of pneumococci—eight more than comparable vaccines—and is priced at around $300 per dose.

Additionally, CDC experts noted that Black Americans tend to experience pneumococcal illness earlier, typically between 55 and 59 years of age, which influenced the decision to lower the initial vaccination age.

The CDC also indicated that booster doses may be needed approximately 15 years after the initial vaccination. Hospital pharmacists can play a critical role in educating patients about this updated recommendation, ensuring those eligible receive timely vaccinations to prevent serious infections.

NORTH CHICAGO, IL — AbbVie recently announced that the U.S. Food and Drug Administration (FDA) has approved VYALEV™ (foscarbidopa and foslevodopa) as the first and only subcutaneous 24-hour infusion of levodopa-based therapy for the treatment of motor fluctuations in adults with advanced Parkinson's disease (PD).

"For too long, the Parkinson's community has had limited treatment options for advanced disease. Due to the progressive nature of the disease, oral medications are eventually no longer as effective at motor symptom control and surgical treatment may be required," said Robert A. Hauser, M.D., MBA, Professor of Neurology and Director of the Parkinson's and Movement Disorder Center at the University of South Florida. "This new, non-surgical regimen provides continuous delivery of levodopa morning, day and night."

The approval was supported by the pivotal Phase 3, 12-week study evaluating the efficacy of continuous subcutaneous infusion of VYALEV in adult patients with advanced PD compared to oral immediate-release carbidopa/levodopa (CD/LD IR), along with a 52-week, open-label study which evaluated the long-term safety and efficacy of VYALEV.

Findings from the pivotal study showed patients receiving VYALEV demonstrated superior improvement in motor fluctuations, with increased "on" time without troublesome dyskinesia and decreased "off" time, compared with oral CD/LD IR. "On" time refers to the periods of time when patients are experiencing optimal motor symptom control while "off" time is when symptoms return.

The majority of adverse reactions (ARs) with VYALEV were non-serious and mild or moderate in severity. The most frequent ARs (greater than or equal to 10 percent and greater than CD/LD IR incidence) were infusion site events, hallucinations, and dyskinesia.

"People living with advanced Parkinson's disease experience daily challenges as a result of uncertainty in managing motor fluctuations, especially as their disease progresses," said Roopal Thakkar, M.D., executive vice president, research & development, and chief scientific officer, AbbVie. "We are proud to bring this innovation to patients who may benefit from motor symptom control through continuous 24-hour administration of VYALEV."

PD is a progressive and chronic movement disorder resulting in tremor, muscle rigidity, slowness of movement and difficulty with balance resulting from the loss of dopamine-producing brain cells.

Timing for a patient's access to VYALEV is dependent on their individual insurance plan. Coverage for Medicare patients is expected in the second half of 2025.  

SAVANNAH, GA - Johnson & Johnson recently announced positive results from the Phase 2/3 Vibrance-MG study of nipocalimab in anti-AChR positive adolescents (aged 12 – 17 years) living with generalized myasthenia gravis (gMG). Study participants who were treated with nipocalimab plus standard of care (SOC) achieved sustained disease control as measured by the primary endpoint of immunoglobulin G  (IgG) reduction from baseline over 24 weeks, and secondary endpoints of improvement in MG-ADLand QMG scores. These Phase 2/3 data will be featured in an oral presentation (Abstract #MG100) at the Myasthenia Gravis Foundation of America (MGFA) Scientific Session during the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, where Johnson & Johnson will present 25 abstracts.  

"Findings from the Vibrance-MG study underscore the potential of this investigational therapy for young individuals aged 12 – 17 living with gMG. Results show a significant reduction in IgG of approximately 70% in adolescents and a clinical benefit that is consistent with the Vivacity-MG3 study in adults," said Jonathan Strober, M.D., Director of Clinical Services for Child Neurology and Director of the Muscular Dystrophy Clinic at UCSF Benioff Children's Hospital.^d "It is encouraging to see these positive results as there are currently no approved advanced treatment options for this adolescent population in the United States."

About 10% of new cases of myasthenia gravis are diagnosed in adolescents (12 – 17 years of age) and the severity of gMG in pediatric patients is heightened with 43% having experienced over five hospitalizations in their lifetime, 46% having at least one intensive care unit stay and 68% having periods of exacerbated disease.

Treatment with nipocalimab plus SOC met the study's primary endpoint of reduction in total serum IgG (-69%), and the two secondary endpoints of MG-ADL and QMG, which are measures of disease activity. Four of five patients achieved minimum symptom expression (MG-ADL score 0-1) by the end of their treatment phase. Nipocalimab was well-tolerated over the six-month period, similar to tolerability seen in adult participants in the Vivacity-MG3 study. There were no serious adverse events and no discontinuations due to an adverse event.

Presented for the first time, these open-label Phase 2/3 results in adolescents are consistent with findings from the pivotal study of nipocalimab in adult patients with gMG. Nipocalimab when added to SOC is the first FcRn blocker to demonstrate sustained disease control in a registrational trial as measured by improvement in MG-ADL over placebo plus SOC over a period of six months of consistent dosing (Q2 week) among adults living with gMG.

"The Vibrance-MG data add to the expanding clinical profile of nipocalimab and highlight its potential for adolescents living with gMG who are in need of new treatments," said Sindhu Ramchandren, M.D., Executive Medical Director, Neuroscience, Johnson & Johnson Innovative Medicine. "We are committed to developing innovations for autoantibody-driven neurological diseases, like gMG, with the aim of transforming the lives of people living with these conditions."

Earlier this year, Johnson & Johnson announced the submission of applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) seeking approval for nipocalimab for the treatment of gMG. 

A recent study suggests that Medicare could significantly reduce waste from the Alzheimer’s drug lecanemab by introducing a new vial size, potentially saving up to 74% of the $336 million lost annually due to discarded medication. The research, published on October 14 in JAMA Internal Medicine, highlights the issue of drug wastage caused by dosage discrepancies and offers a straightforward solution: adding a 75 mg vial to complement the existing 500 mg and 200 mg vials.

Lecanemab, a drug used to treat people with mild cognitive impairment or mild dementia, is currently packaged in single-use vials. Since dosages are based on patients' body weight, the one-size-fits-all packaging leads to significant wastage when the prescribed dose is lower than the vial size. For example, a patient requiring 650 mg would receive one 500 mg and one 200 mg vial, leaving 50 mg of unused and discarded medication.

Lead author Frank Zhou, a medical student at the David Geffen School of Medicine at UCLA, emphasized the urgency of reducing such wasteful spending. “It is imperative to reduce spending on services that do not improve the health of patients, and this is a prime example,” Zhou said, noting that part of the problem is that Medicare is paying for drugs that are quite literally being thrown away.

The study's senior author, Dr. John Mafi, associate professor of medicine at UCLA, noted that Medicare spent $33 billion on Part B infusion drugs in 2021, a category that includes lecanemab. Given that waste from just this one drug could cost hundreds of millions, Mafi believes there is "substantial opportunity for cost savings by reducing waste from all infusion drugs."

The study comes as healthcare costs continue to rise, placing increasing pressure on Medicare’s budget. Medicare is already mandated to seek reimbursements from manufacturers for waste exceeding 10% under the 2021 Infrastructure Investment and Jobs Act. However, the researchers estimate that lecanemab waste is only 5.8%, falling short of the current reimbursement threshold.

The study's findings were based on data from the 2020 Health and Retirement Study, which involved participants aged 65 and older with Medicare Part B coverage. Researchers calculated individual weight-based doses and compared them to the dispensed dose, estimating annual drug waste. The results showed that lecanemab waste could provide treatment for an additional patient for every 16 patients treated under current vial sizing.

While the proposed 75 mg vial would reduce waste by 74%, the researchers acknowledge some limitations in their study. These include potential differences in weight distribution between the study population and actual lecanemab users, uncertain uptake rates, and the exclusion of manufacturing and regulatory costs in the analysis.

The research team includes experts from UCLA and the RAND Corporation, with funding from the National Institute on Aging. The study underscores the need for further policy changes to address drug waste and reduce healthcare costs, potentially opening the door for similar savings across other infusion therapies.

SOURCE: UCLA, news release, Oct. 14, 2024

Philadelphia, PA – GSK has released promising new data from the AReSVi-006 Phase III trial, which evaluated the efficacy and safety of a single dose of AREXVY, the world’s first Respiratory Syncytial Virus (RSV) vaccine, in preventing lower respiratory tract disease (LRTD) caused by RSV in adults aged 60 and older. The data, spanning three full RSV seasons, includes results for adults at increased risk due to underlying health conditions.

The trial results show that a single dose of AREXVY provided a clinically meaningful cumulative efficacy of 62.9% against RSV-related LRTD (97.5% CI, 46.7-74.8) and 67.4% against severe RSV-LRTD (95% CI, 42.4-82.7) across three RSV seasons. In the third season alone, the vaccine demonstrated 48.0% efficacy against RSV-LRTD (95% CI, 8.7-72.0). These findings are based on the analysis of data from 24,966 adults aged 60 years and older.

Safety and reactogenicity findings were consistent with earlier data from the Phase III program, confirming the vaccine was generally well tolerated. The most common adverse events reported within four days of vaccination included injection site pain, fatigue, muscle aches, headache, and joint pain.

AREXVY's efficacy was consistent across various RSV subtypes and populations, including adults aged 70-79 and those with underlying medical conditions such as COPD, asthma, and chronic heart failure, which place them at greater risk for severe RSV outcomes. Given that RSV can exacerbate these conditions and lead to severe health complications, the vaccine's protection over three seasons is expected to significantly impact public health.

“The potential health impact of a single dose of AREXVY protecting older adults for three RSV seasons is substantial, especially given the increased risk that RSV poses to this population,” said Tony Wood, Chief Scientific Officer at GSK. “This is the only RSV vaccine with safety and efficacy data extending over three seasons. We remain committed to sharing further data on long-term protection and revaccination schedules to support public health decisions.”

RSV is a highly contagious virus that affects the lungs and respiratory passages, with an estimated 64 million people globally contracting RSV each year. Adults, particularly those with comorbidities or compromised immune systems, face heightened risks of severe outcomes, including hospitalization and death. In the U.S. alone, approximately 177,000 adults aged 65 and older are hospitalized annually due to RSV, with an estimated 14,000 deaths resulting from these cases.

GSK will continue to share long-term data on efficacy, immune response, and revaccination to inform future immunization guidelines. The results highlight the potential of AREXVY to provide year-round protection for older adults and help prevent RSV-related complications.

The Centers for Disease Control and Prevention (CDC) is informing public health officials, clinicians, and affected patients, their families, and caregivers about a potential public health risk among individuals ordering what they believe to be prescription medications from online pharmacies. On September 30, 2024, the U.S. Department of Justice (DOJ) announced an indictment against individuals running illegal online pharmacies. According to the DOJ, these individuals are advertising, selling, manufacturing, and shipping millions of unregulated counterfeit prescription pills to tens of thousands of individuals in the U.S. The counterfeit pills frequently contain fentanyl, a synthetic opioid that is up to 50 times stronger than heroin and 100 times stronger than morphine, and methamphetamine. Individuals who ordered drugs from these illegal pharmacies could be at risk for an overdose.

The U.S. Food and Drug Administration warns that there are online pharmacies that claim to sell prescription drugs at deeply discounted prices, often without requiring a prescription. These internet-based pharmacies often sell unapproved, counterfeit, or otherwise unsafe medicines outside the safeguards followed by licensed pharmacies. Individuals should only take medications prescribed by a licensed healthcare provider and dispensed by a licensed pharmacy.

According to the National Association of Boards of Pharmacy, nearly 95% of websites offering prescription-only drugs online operate illegally.

Paris and Tarrytown, NY,  - The US Food and Drug Administration (FDA) has approved Dupixent (dupilumab) as an add-on maintenance treatment of adults with inadequately controlled chronic obstructive pulmonary disease (COPD) and an eosinophilic phenotype. Dupixent is the first biologic medicine approved in the US to treat these patients.

“People living with inadequately controlled COPD have long awaited new medicines to help manage the daily suffering they experience from breathlessness, coughing, wheezing, exhaustion and unpredictable hospitalization. These patients often struggle with everyday activities many people take for granted such as taking a walk or running errands outside the home. We welcome the approval of this new therapeutic option to offer patients a new way to help gain better control of their disease,” said Jean Wright, M.D., Chief Executive Officer at The COPD Foundation.

The FDA approval is based on data from two landmark pivotal phase 3 studies (BOREAS and NOTUS) that evaluated the efficacy and safety of Dupixent compared to placebo in adults currently on maximal standard-of-care inhaled therapy (nearly all on triple therapy) with inadequately controlled COPD and blood eosinophils ≥300 cells per μL. Patients who received Dupixent in BOREAS (n=468) and NOTUS (n=470) experienced the following outcomes, respectively, compared to placebo (BOREAS n=471; NOTUS n=465):

• 30% and 34% reduction in the annualized rate of moderate or severe COPD exacerbations over 52 weeks, the primary endpoint.
• 74mL and 68mL numerically greater improvements in post-bronchodilator FEV₁ from baseline at week 12 compared to placebo, sustained at 52 weeks. Statistically significant improvements of similar magnitude were observed in pre-bronchodilator FEV₁ from baseline at 12 and 52 weeks, a key secondary endpoint.
• 51% response in a health-related quality of life measure in both trials compared to 43% and 47% with placebo at 52 weeks, as assessed by a 4-point improvement on the St. George’s Respiratory Questionnaire (SGRQ).        
   

Safety results in both studies were generally consistent with the known safety profile of Dupixent in its approved indications. In pooled BOREAS and NOTUS data, the most common adverse events (>2%) more frequently observed in patients on Dupixent compared to placebo were viral infection, headache, nasopharyngitis, back pain, diarrhea, arthralgia, urinary tract infection, local administration reaction, rhinitis, eosinophilia, toothache, and gastritis. While less common, cholecystitis was reported in 0.6% of patients on Dupixent compared to 0.1% of patients on placebo.

The FDA evaluated Dupixent under Priority Review, which is reserved for medicines that represent potentially significant improvements in efficacy or safety in treating serious conditions. In July 2024, Sanofi and Regeneron announced that the European Medicines Agency approved Dupixent as an add-on maintenance treatment for adults with uncontrolled COPD characterized by raised blood eosinophils. Submissions are currently under review with other regulatory authorities around the world, including in Japan.