JnJ gets USFDA nod for Opsynvi for chronic treatment of adults with pulmonary arterial hypertension

Raritan: Johnson & Johnson has announced that the U.S. Food and Drug Administration (FDA) has approved OPSYNVI – a single-tablet combination of macitentan, an endothelin receptor antagonist (ERA), and tadalafil, a phosphodiesterase 5 (PDE5) inhibitor – for the chronic treatment of adults with pulmonary arterial hypertension (PAH, World Health Organization [WHO] Group I) and WHO functional class (FC) II-III. 

OPSYNVI may be used in patients with PAH who are treatment-naïve or who are already on an ERA, PDE5 inhibitor or both. OPSYNVI may be used in patients who are currently treated concomitantly with stable doses of macitentan 10 mg and tadalafil 40 mg (20 mg x 2) as separate tablets.

PAH is a rare, progressive and life-threatening blood vessel disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation that eventually leads to right heart failure. An estimated 500 to 1,000 new cases of PAH are diagnosed each year in the U.S., classifying the disease as a rare condition.

The 2022 European Society of Cardiology (ESC) / European Respiratory Society (ERS) clinical guidelines recommend initial combination therapy of an ERA and a PDE5 inhibitor for patients with idiopathic PAH, heritable drug-associated PAH, or PAH-associated with connective tissue disease without cardiopulmonary comorbidities at low or intermediate risk.

“Clinical guidelines recommend treating patients with initial and sequential dual-combination therapy, regardless of risk at initial diagnosis and follow-up. Historically, this required patients to take multiple pills because no single-tablet combination therapy targeting two or more pathways was available,” said Kelly Chin, M.D., Professor of Internal Medicine and Director of the Pulmonary Hypertension Program at UT Southwestern Medical Center, and an investigator in the A DUE study.* “As administration of macitentan and tadalafil together are commonly prescribed for initial therapy for PAH, the introduction of a single tablet combining both is promising for clinicians treating patients as it may help bridge the gap between clinical guidelines and everyday clinical practice, while offering a patient-friendly approach to support initial combination therapy and rapid escalation for the appropriate patients.”

The FDA’s approval of OPSYNVI is based on the results from the pivotal Phase 3 A DUE study, in which OPSYNVI demonstrated greater reduction in Pulmonary Vascular Resistance (PVR) after 16 weeks versus tadalafil or macitentan monotherapy. OPSYNVI has a Boxed Warning due to the risk of embryo-fetal toxicity and requires female patients to enroll in the Macitentan-Containing Products Risk Evaluation and Mitigation Strategy (REMS) program.

With the approval, Johnson & Johnson now offers a PAH portfolio addressing all three foundational and guideline-recommended pathways – nitric oxide, endothelin, and prostacyclin.

“People with PAH often live with the burden of taking many pills each day, which can pose challenges,” said James F. List, M.D., Ph.D., Global Therapeutic Area Head, whose team oversees a portfolio of programs including Pulmonary Hypertension at Johnson & Johnson. “We’re thrilled to bring this single tablet combination therapy to patients, as it has the potential to optimize disease management and fulfill a significant unmet need in supporting recently updated treatment guidelines that call for initial or early combination treatment.”

Individually, macitentan reduces the risk of clinical worsening events and hospitalization, and tadalafil improves exercise ability.

The A DUE study was a double-blind, randomized, active-controlled, multi-center, adaptive, parallel-group study designed to compare the efficacy and safety of OPSYNVI to macitentan and tadalafil monotherapies in adult patients with PAH (WHO FC II or III). The three-arm trial enrolled patients from across 76 sites in 16 countries/territories worldwide who were treatment-naïve or on a stable dose of an endothelin receptor antagonist (ERA), or a phosphodiesterase 5 (PDE5) inhibitor, for at least three months. The primary endpoint was change from baseline in PVR at the end of double-blind treatment at 16 weeks and was considered met if macitentan and tadalafil fixed-dose combination (FDC) treatment was superior to both monotherapies. Following the treatment period, patients transitioned to the open-label treatment period for 24 months.

Read also: USFDA nod to Johnson and Johnson EDURANT PED for certain pediatric patients living with HIV-1

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New classification of tuberculosis to support efforts to eliminate the disease

A new way to classify tuberculosis (TB) that aims to improve focus on the early stages of the disease has been presented by an international team involving researchers at UCL.

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Eligibility for lung cancer screening up with 2021 USPSTF recommendations

Expanded U.S. Preventive Services Task Force (USPSTF) criteria for lung cancer screening (LCS) in 2021 have resulted in a 65.9 percent increase in the number of eligible individuals, according to a research letter published online March 21 in JAMA Network Open.

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Survey assesses perspectives on patient image use in dermatology

Most patients report an increase in trust in the quality of their medical care if asked for permission to use their images for research or education, according to a research letter published online March 13 in JAMA Dermatology.

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Systemic inflammation increases risk for chronic kidney disease

Systemic inflammation is associated with an elevated risk for chronic kidney disease, according to a study published online Feb. 20 in Frontiers in Immunology.

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Study: For each 10% increase of bacteria type in the gut, risk of hospitalization for infection falls by up to a quarter

A study of two large European patient cohorts has found that for every 10% increase in butyrate-producing bacteria in a patient’s gut, the risk of hospitalization for any infection falls by between 14 and 25% across two large national cohorts. The study will be presented at this year’s European Congress of Clinical Microbiology and Infectious Diseases (ECCMID 2024) in Barcelona, Spain (27–30 April).

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Violation of PC PNDT Act: Licence of three ultrasound centres suspended, show cause notice issued

Ludhiana: In violation of the Pre-Conception and Pre-Natal Diagnostic Techniques (PC-PNDT) Act 1994, the state health department suspended the licences of three ultrasound centres and issued show-cause notices to two such centres. 

The action was taken following an inspection that revealed numerous irregularities and illegal activities in the hospitals/centres. Dr Jasbir Singh Aulakh, the civil surgeon of Ludhiana who carried out the inspections last week, discovered that the centres violated the PC-PNDT Act 1994. Consequently, stringent measures were implemented to uphold the enforcement of the PC-PNDT Act. 

These three health centres/hospitals located at Jagraon, Raikot and Maachiwara came under the scanner of the health department which inspected around 15 ultrasound centres in Samrala, Maachiwara, Jagraon and Raikot on March 18 and 19 under the leadership of Hatur Senior Medical Officer Dr Varun Saggar and Sahnewal SMO Dr Ramesh. 

Although ultrasound machines were seized and the licenses have been suspended at these three facilities, the health department also issued a show cause notice to two such centres in Jagraon and Machiwara asking them to submit a response in three days.   

Also read- Illegal Sex Determination Racket Busted In Gujarat, Doctor Arrested

On March 18, Dr Jasbir inspected a facility at Jagraon and found that it was running with a licence for echocardiography only, the scanner was used for pelvic scans. Moreover, the records of the patients who got tested at this facility were not found on OPD records. As a result, the authorities suspended the licence of the hospital and sealed the ultrasound sonography machine until further notice under Section 20(3) of the PC-PNDT Act. The centre received a show-cause notice and was given a three-day deadline to respond.

On March 19, a second hospital in Raikot was inspected where it was found that the facility failed to maintain a proper record of patients under the Act. The form F, which includes patient details, the indication of the test, the patient’s declaration of not wanting to know the sex of the fetus, and the doctor’s declaration of not determining/conveying the sex of the fetus, was not filled out. Additionally, the ultrasound machine in this facility was sealed under Section 20(3) of the Act.

In another hospital in Machhiwara, Dr Jasbir found that the facility was involved in shady practices as some forms were found cancelled at that time. Therefore, the authorities issued a show cause notice to this facility and asked them to submit a response within two days, HT reports.

It was discovered that the in-charge doctor was out of the country without informing the medical authority concerned. The keys to the centre were with the staff. The machines were sealed and the licence was cancelled under Section 17(4) of the Act.

Also read- Haryana Doctor, Repeat Offender Under PNDT Act Absconding After Another Raid

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Understanding Chemotherapy: A Comprehensive Guide to Treatment Plans – Dr Philip George Kuttikat

Chemotherapy is a cornerstone in the battle against cancer, serving as a vital treatment modality. This multifaceted strategy employs potent drugs to target and eradicate rapidly dividing cancer cells throughout the body. Despite its demanding nature, chemotherapy significantly contributes to managing various cancer types and enhancing patients’ survival rates.

Mechanism of Action
Chemotherapy operates by disrupting the cell cycle, hindering cancer cells from proliferating and dividing. Unlike targeted therapies, which focus on specific cancer-associated molecules, chemotherapy has a broader impact, affecting both cancerous and healthy cells.
This non-selective characteristic contributes to its associated side effects, impacting healthy, rapidly dividing cells such as those found in the bone marrow and digestive tract.

Types of Chemotherapy
Chemotherapy can be administered through various means, including intravenous injections, oral medications, and topical creams. The selection of chemotherapy drugs and their delivery methods depends on factors such as cancer type, stage, and patient health.

Additionally, considerations like age, overall health, and existing medical conditions play pivotal roles in determining the appropriate chemotherapy regimen. Combination chemotherapy, utilizing multiple drugs, is often utilized to enhance effectiveness and mitigate drug resistance risks.

Chemotherapy Agents 
• Alkylating Agents: Examples include cyclophosphamide and cisplatin.
• Antimetabolites: Examples include methotrexate and 5-fluorouracil.
• Topoisomerase Inhibitors: Examples include etoposide and irinotecan.
• Mitotic Inhibitors: Examples include paclitaxel and vinblastine.
• Targeted Therapies: These encompass hormone therapy, monoclonal antibodies, tyrosine kinase inhibitors, and immunotherapies.

Side Effects 

Chemotherapy-induced side effects, though well-documented, can pose significant challenges for patients. Common adverse effects include fatigue, nausea, hair loss, and weakened immunity. Typically, these effects are transient and resolve following treatment completion.

Many patients may require injections to boost blood counts and oral antimicrobial medications to manage immune suppression.

Combination Therapies

In certain cases, chemotherapy is combined with other treatment modalities, such as surgery or radiation therapy.

This integrated approach, known as multimodal or combination therapy, aims to maximize treatment efficacy by addressing cancer from multiple angles. The synergy between these treatments often leads to improved outcomes for specific cancers.

Role of Chemotherapy in Different Cancer Stages

1. Early Stage (Stages 0 to II)

• Surgery or localized treatments like radiation therapy may be primary interventions when cancer is localized and hasn’t extensively spread.
• Adjuvant chemotherapy post-surgery helps eliminate residual cancer cells, reducing recurrence risks.
• Neoadjuvant chemotherapy pre-surgery shrinks tumours, facilitating surgical removal.

2. Locally Advanced Stage (Stage III) 

• Combination therapy involving surgery, radiation, and chemotherapy may be employed for cancers spreading to nearby tissues or lymph nodes.
• Neoadjuvant chemotherapy may precede surgery or radiation to improve local control.
• Adjuvant chemotherapy post-surgery or radiation targets residual cancer cells, reducing recurrence risks.

3. Metastatic or Advanced Stage (Stage IV) 

• Systemic treatments like chemotherapy become primary approaches when cancer spreads to distant organs or lymph nodes.
• Palliative chemotherapy aims to alleviate symptoms, shrink tumors, and enhance quality of life.
• Targeted therapies and immunotherapies may complement chemotherapy based on cancer characteristics.

4. Recurrent or Resistant Disease

• Chemotherapy options are reassessed for recurrent or resistant cancers.
• Different chemotherapy drugs or combinations are considered based on specific cancer characteristics.
• Targeted therapies, immunotherapies, or clinical trial participation may be explored for advanced or recurrent cases.

Despite its demands, chemotherapy embodies the resilience of individuals battling cancer. It’s a journey marked by endurance, triumphs, and moments of resilience that deserve acknowledgement.

On World Cancer Day, observed on February 4th, let’s extend a powerful message of hope, strength, and solidarity to all those navigating the challenging path of chemotherapy.

Disclaimer: The views expressed in this article are of the author and not of Medical Dialogues. The Editorial/Content team of Medical Dialogues has not contributed to the writing/editing/packaging of this article.

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Zerlasiran shows beneficial outcomes for cardiovascular disease reveals phase 2 study

Researchers have announced a promising 36-week topline data from its phase 2 ALPACAR-360 study which evaluating the efficacy of zerlasiran in patients at high risk of atherosclerotic cardiovascular disease (ASCVD). Zerlasiran, Silence’s pioneering siRNA (short interfering RNA) therapy targets the reduction of lipoprotein(a) or Lp(a) as a critical genetic risk factor for cardiovascular diseases that affect a significant portion of the global population.

This comprehensive study included a total of 178 subjects with increased levels of lipoprotein(a), or Lp(a), a genetic factor known to contribute to cardiovascular disease in up to 20% of the global population. This double-blind, placebo-controlled phase 2 trial administered zerlasiran at doses of 300 mg every 16 or 24 weeks and 450 mg every 24 weeks to participants, who had a median baseline Lp(a) of approximately 215 nmol/L. Also, the study reported a median reduction in Lp(a) levels of 90% or greater at the 36-week mark when compared to the placebo group, with no new safety concerns emerging.

The ongoing 60-week study will further assess secondary endpoints of changes in Lp(a) levels from baseline to 48 and 60 weeks, along with potential impacts on other lipid profiles. These findings bolster the potential of zerlasiran as a transformative treatment for patients with high Lp(a) by addressing a significant unmet need in cardiovascular disease management.

The Head of Research and Development at Silence, Dr. Steven Romano, expressed enthusiasm about the phase 2 results marking their consistency with phase 1 outcomes and the competitive profile of zerlasiran for treating high Lp(a) levels. The company anticipates the release of further data from the ALPACAR-360 study in the coming months with the hopes to advance zerlasiran towards regulatory approval and commercial availability.

Silence Therapeutics stands at the forefront of RNA interference (RNAi) technolog and is constantly developing a new class of precision medicines aimed to silence genes implicated in various diseases. Its proprietary mRNAi GOLD™ platform facilitates the creation of siRNAs targeting disease-associated genes in the liver that offer a broad therapeutic potential. Along with zerlasiran, the pipeline includes divesiran for hematological conditions and ongoing collaborations with industry leaders like AstraZeneca and Hansoh Pharma. While the future of zerlasiran looks promising, Silence Therapeutics cautions the investors and stakeholders about forward-looking statements by illuminating the inherent risks and uncertainties in drug development and approval processes.

Source:

(N.d.-a). Silence-therapeutics.com. Retrieved March 22, 2024, from https://silence-therapeutics.com/investors/press-releases/press-releases-details/2024/Silence-Therapeutics-Announces-Positive-Topline-36-Week-Data-from-Ongoing-Phase-2-Study-of-Zerlasiran-in-Patients-with-High-Lipoproteina/default.aspx

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KIMS Bhubaneswar doctor saves woman mid-air

Bhubaneswar: In a remarkable display of quick thinking and medical expertise, Dr Ashok Kumar Badamali, senior consultant in Cardiac Anesthesia at Kalinga Institute of Medical Sciences (KIMS) Bhubaneswar, saved the life of a fellow passenger aboard Air India Express flight I5 764 enroute from New Delhi to Pune. 

The incident, which unfolded in the early hours of the morning, began when the cabin crew, led by Mr. Saurav, made an urgent announcement requesting the assistance of any onboard paramedics or nursing personnel due to a medical emergency.

A middle-aged woman was found unresponsive in her seat, prompting immediate action. Dr Badamali, who was traveling to Pune for research activities, promptly responded to the call for help. The patient was moved to the floor of the aircraft to facilitate resuscitation efforts. Upon examination, Dr. Badamali found the patient to be unresponsive, and exhibiting agonal breathing, with an extremely fast carotid pulse. Utilizing his expertise in cardiac emergencies, he performed a carotid massage that proved to be life-saving. 

Also Read:Doctors at KIMS Health perform carotid revascularization procedure to remove 10-cm-long occluded blockage

Under Dr Badamali’s careful management, the patient was placed in the recovery position and remained stable until the flight successfully landed at Pune International Airport. This act of quick thinking and medical intervention highlights the critical importance of having medical professionals readily available and able to respond in flight emergencies.

Reflecting on the incident, Dr Badamali noted a few constraints in the current emergency response protocols aboard flights. He said that he will draft and send a Standard Operating Procedure (SOP) to the Aviation Ministry to enhance the management of such medical emergencies in the future, aiming to improve the safety and well-being of passengers.

The pilot of the flight, Mr. Rahul, along with crew members Mr. Saurav and Miss Puja, expressed their profound gratitude to Dr Badamali for his timely and decisive action. They assured him that an incident report would be submitted to the concerned authorities to document the event and the effective response.

Also Read:KIMS Hospital doctors successfully remove massive chest tumour from 25-year-old woman

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