oHCM is a rare disease that occurs when the heart muscle thickens, mostly in the septum (the heart wall that separates its right and left sides) and obstructs blood flow from the heart to the rest of the body. The severity of oHCM varies, but it can cause serious symptoms and death. Symptoms include palpitations (the sensation the heart is pounding), shortness of breath, swelling of the legs, and decreased exercise capacity, which significantly impact daily activities and quality of life.
Camzyos works by reducing contraction of the heart muscle, which can cause heart failure or totally block functioning of the ventricles (lower chambers of the heart). Patients who have a serious intercurrent illness (such as a serious infection) or arrhythmia (atrial fibrillation or other uncontrolled fast heart rhythm abnormality) are at greater risk of developing impaired heart muscle contraction and heart failure with Camzyos.
Because of the risk for heart failure, patients treated with Camzyos must be monitored with echocardiograms, an imaging test that shows how well the heart is working. Patients must also avoid certain prescription and over-the-counter medicines that interfere with the metabolism (breakdown) of Camzyos. At the end of March 2022, the FDA approved Ztalmy (ganaxolone) to treat seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older. This is the first treatment for seizures associated with CDD and the first treatment specifically for CDD. CDD is a rare developmental epileptic encephalopathy (dysfunction of the brain) caused by CDKL5 gene mutations. The CDKL5 gene is responsible for making proteins that are important for normal brain functioning and development. Patients with CDD typically have infantile-onset epilepsy that responds poorly to currently available treatments. Other symptoms include hypotonia (poor muscle tone), severe developmental and cognitive delays with little or no speech production, fine and gross motor impairment (including inability to walk for most patients), cortical visual impairment, behavioral abnormalities, and sleep and digestive difficulties. Although rare, the incidence of CDD is believed to be between 1 in 40,000–60,000 live births, making it one of the most common genetic forms of epilepsy.
Covid treatments continue to make good strides. April 2022 saw the FDA approve the COVID-19 treatment Veklury (remdesivir) to include pediatric patients 28 days of age and older weighing at least 3 kilograms (about 7 pounds) with positive results of direct SARS-CoV-2 viral testing, who are:
- Hospitalized, or
- Not hospitalized and have mild-to-moderate COVID-19 and are at high risk for progression to severe COVID-19, including hospitalization or death
This action makes Veklury the first approved COVID-19 treatment for children less than 12 years of age. As a result, the agency also revoked the emergency use authorization for Veklury that previously covered this pediatric population.
Before now, Veklury was approved to treat only certain adults and pediatric patients (12 years of age and older who weigh at least 40 kilograms, which is about 88 pounds) with COVID-19.
To date, the FDA has approved two vaccines, and three vaccines are available for emergency use, to prevent COVID-19 and the serious clinical outcomes associated with COVID-19, including hospitalization and death.
In the UK, the MHRA has approved the COVID-19 vaccine developed by Valneva after a rigorous review as well as Evusheld (tixagevimab/cilgavimab) for COVID-19 prevention, after it had found that it had met the required safety, quality, and effectiveness standards.
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Amritpal Singh Chohan, Newsletter Subcommittee Member
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