Nov 19 (Reuters) – BioMarin Pharmaceutical Inc’s (BMRN.O) once-daily injection for children with the most common type of dwarfism received clearance from the U.S. health regulator on Friday, making it the first approved therapy for achondroplasia in the country.
The approval for Voxzogo, intended for children aged five and older, comes months after it became the first therapy to be greenlighted by regulators for the treatment of the genetic condition in Europe.
Many countries still do not have an approved therapy for Achondroplasia, a condition characterized by short stature which occurs in about one in every 25,000 newborns worldwide.
BioMarin expects the therapy’s annual net revenue per patient in the United States to be about $240,000 and expects about 3,000 children with achondroplasia in the country to be eligible for the treatment. Voxzogo is expected to be available in the United States by mid-to-late December at a list price of $899 per vial, the company said.
BioMarin’s late-stage Voxzogo study showed improvements in patients’ growth rate over a year and was well tolerated. The drug is also being studied in children below five years and has already been approved in Europe for those aged two and above.
The U.S. Food and Drug Administration had in April extended the review of the drug by three months to assess two-year data on the drug.
It has now cleared the drug under the accelerated approval pathway, which requires a post-marketing study assessing final adult height.
BioMarin said it intended to use the results from ongoing extension studies to receive final approval.
The drug is also under regulatory review in Japan, Brazil, and Australia and could receive approval in the countries next year, BioMarin said.
Reporting by Amruta Khandekar; Editing by Aditya Soni and Vinay Dwivedi
Our Standards: The Thomson Reuters Trust Principles.
Keeping pets protected during heatwaves this summer
We’ve been lucky to have been hit with some great weather this summer, however, some days have been exceptionally hot and causing us discomfort to the point where we don’t want to leave the house! Our pets can also struggle in this heat and if not looked after correctly, can lead to life threatening issues for them.
Dr. Sarah Machell, Medical Director for Vetster, has shared her top five top tips to ensure that our pets can enjoy the summer days as much as we can.
Vetster, is a digital platform that connects licensed vets with pet owners virtually, with 24/7 online appointments, launches in the UK this summer.
1. Provide adequate rest, shade, and ventilation
Coping with high temperatures and humidity is tough enough on its own, but it’s even more difficult for pets who are exercising in direct sunlight and don’t have adequate ventilation. Limit outdoor activities to early mornings and late evenings when it’s cooler. When walking your pet, choose shady routes off the pavement. Ensure outdoor pets have shady, ventilated places to escape the heat. Keep in mind that pets also rely on evaporation for cooling, regardless of if they sweat like horses or pant like dogs, and high humidity decreases the effect of evaporation. This means your pet needs you to keep an eye on the heat index for them as well as for you. Be sure your pets have easy access to a steady supply of clean water. Pets are naturally wired to stay hydrated as long as they are healthy and avoid heavy exercise in the heat, so there’s no need to try to encourage your pet to drink more. Just make sure the water supply is in easy reach and doesn’t run dry.
2. Be wary of paws on the hot pavement!
When the weather gets extremely hot, so does the pavement—asphalt, in particular. If you’re taking your dog out for a walk, try to remember that they don’t have shoes to protect against the heat. Even though paw pads are extremely tough, hot surfaces can burn them. Consider using padded booties for their paws to create a barrier between paw pads and the hot concrete. Better yet, try to steer clear of the streets and walk on the cool grass instead. If there’s an opportunity to wade through some clean water or catch the spray from a sprinkler, that’s even better. Remember that pavement retains heat and you still need to be aware of the risks when you go for walks in the evening.
3. Look out for signs of heatstroke
Heatstroke is a very serious condition and one to look out for in your pets. As a pet parent, it’s important to be aware of your pet’s fitness level and avoid overexertion when the weather’s too hot or humid. Less athletic dogs, dogs with underlying illnesses, and brachycephalic breeds are at higher risk for developing heatstroke, so keep an extra close eye on them. Heatstroke is life-threatening, but it can be avoided if you take action to cool your pet when they show early signs of heat stress. If your dog doesn’t want to keep walking, lies down in the shade, or digs up cool dirt to lie in, those are clues they’re getting too hot. Excessive panting that doesn’t improve after a short rest is another indicator. Get out of the heat and offer water to keep the threat of heatstroke from escalating. Splash down hot ears, paws, and bellies with water to achieve more rapid cooling.
4 Never leave your pet unattended in a hot vehicle
Heatstroke can happen in the blink of an eye—it cannot be stressed enough that you should never, ever leave your pet unattended in a vehicle. This is true even if you leave the windows down for fresh air or if you think you’ll only be gone for a few minutes. Studies show that even if the outdoor temperature is 72℉ (22°C), a car can rise up to 117℉ (47°C) in only an hour. Imagine how quickly a car can become dangerously hot when outdoor temperatures are a balmy 86℉ (30°C). Even if you’re leaving your car unattended for a minute or think that leaving a window open will help – the life-or-death gamble you’re taking isn’t worth it.
Why dosage matters in menopause treatment
By Rizvan Faruk Batha MPharm, PGDip GPP, IPresc, MRPharmS, Superintendent Pharmacist of Specialist Pharmacy.
Bioidentical Hormone Restoration Therapy (BHRT) is an alternative option to traditional synthetic HRT, using bioidentical hormones to treat hormonal conditions in both men and women. BHRT is compounded medicine and put simply, means that the individualised ingredients are mixed together under the direction of a qualified prescriber’s prescription to meet the tailored needs of a patient.
For women going through menopause, compounding treatments are often prescribed later down the line, when a woman has been unable to settle on the appropriate dose of HRT with a General Practitioner, often experiencing severe side effects from either too much or too little HRT. In short, compounded menopause medication offers another route for those patients where the licensed preparation is not appropriate or hasn’t worked.
Patient-led care plays an important role in compounded menopause medicine because the patient is involved throughout the process; during the consultation with the prescribing practitioner, and with the pharmacist developing the customised dosage.
One of the biggest challenges faced by compounding pharmacies is the drugs being classified as unlicensed. For drugs to be licensed, it involves research and clinical trials to assess the efficacy, quality, and safety of the medicines, and because of this process, more often than not it is the recommended route to prescribe licensed medicines. That being said, prescribing unlicensed compounded menopause medicines may be necessary, especially when it comes to the patient’s specific need and interests and where licensed medicines have been unable to satisfactorily meet the needs of the patient or are unsuitable for them.
Utilising compounded menopause medicine as a method to enhance patient care could be beneficial for the many patients that need specific dosing or formulation requirements, but sadly more often than not we see delays to patient needs and treatment, as the rise of mass manufactured licensed medicines have grown in popularity. Compounding menopause medicine could offer huge potential for many, but prescribing practices have moved towards evidence-based medicines because of the responsibility imposed on prescribers for prescribing compounded therapies. Clinicians need to understand that even licensed products are not safe or effective for all patients particularly if the product is being used in a population that were not part of the original clinical trials for the drug.
It is important to know that compounding pharmacies and pharmacists in the UK are also regulated and licensed by the General Pharmaceutical Council (GPhC). The GPhC set standards for pharmacists and pharmacies to meet to remain on their register with the aim to protect the public and give them assurance that they will receive safe and effective care when using pharmacy services.
So even though compounded medicines are ‘unlicensed’, there is a lot of due diligence exercised by the pharmacists to ensure the products meet the safe and effective care criteria. This is generally demonstrated through the purchase of medical grade active and inactive pharmaceutical ingredients (with certificates of analysis and safety data sheets), trained staff, following and updating SOPs, audits, traceability of ingredients and products during recalls, as well as continuous learning and error reporting being supported in the pharmacy.
Ultimately, how compounded menopause medicine is viewed will depend on the knowledge and experience of the patients and professionals involved. Although the medicine is unlicensed it is important to understand that experienced clinicians and pharmacists involved in the process of making the decisions are regulated, and patients are consistently monitored during their response to the medicine. If compounded menopause medicine was integrated in the healthcare system, it could change and improve the quality of life of many patients suffering with debilitating menopause symptoms, and we hope as a pharmacy that in the future we will see a shift in how compounded medicine is viewed.
We need more than investment in services to solve the mental health crisis.
By Lea Milligan
It’s now widely recognised that there is a crisis in mental health. In September, a staggering 1.6m people in the UK are waiting for mental health treatment on the NHS, and 1 in 4 people globally are impacted by a mental illness at some point in their life.
This crisis is only being exacerbated by the increasing cost of living, global conflicts causing generational trauma and the ongoing fallout from COVID-19.
Lack of equitable access to services, treatment and resources means the most vulnerable in society continue to suffer disproportionately.
Everyone assumes that addressing this crisis requires more doctors, more appointments and more mental health treatment centres. Whilst one answer is, yes, we absolutely need more of these, it’s not the only answer.
More services are not the sole solution: We also need more research.
Mental health research is the secret weapon that often is overlooked by policy makers.
- Research shows us where to focus resources, so that we don’t just blindly throw money and time at the parts of the system that are not working.
- Research helps move people through the system faster, giving them more effective treatments and faster diagnoses.
- Research reduces the number of people requiring acute care by providing effective prevention strategies and intervention methods.
Research speeds up access to services:
The EnCAMHS project, sponsored by Greater Manchester Mental Health NHS Foundation Trust, is working to improve and refine the referral process to CAMHS (Child and Adolescent Mental Health Services). By mapping and raising awareness among professionals of the different types of support available for children and young people, the EnCAMHS team are hoping to reduce waiting lists and speed up access to care for the most urgent cases.
Research improves treatments:
I have seen firsthand the incredible breakthroughs in treatments that have come from targeted investment in research.
MQ researcher Dr Colette Hirsch from King’s College London developed a new treatment for anxiety and depression called cognitive bias modification for interpretation (CBT-I). New treatments such as this help provide a greater range of treatment choices, more important now than ever because, according to the World Health organisation, the pandemic triggered a 25% increase in anxiety and depression worldwide.
Dr Jennifer Wild from Oxford University has developed a new treatment for PTSD in health care workers, with a 90% recovery rate. This is an unprecedented success rate for a group highly vulnerable to traumatic stress.
And Dr Ethel Mpungu has developed a community-based therapy for people living with both depression and AIDS in rural Uganda. The 578 participants of her study remained symptom free from depression 12 months later. Her work has been internationally recognised and, due to its success and cost-effectiveness, it is being rolled out across other African nations, improving the lives of millions of people.
Research can help us prevent mental ill-health in the first place:
For example, the IDEA (Identifying Depression in Early Adolescence) project has created a prediction model to identify the young people who are most at risk of developing depression in later life. This tool can be used to identify the most vulnerable before depression can take hold, getting them timely interventions that can prevent a lifetime of mental ill-health.
This Mental Health Awareness Week (9-15 May), plenty of people will be talking about smashing stigmas, reducing loneliness and the need for more mental health support. And, of course, these are absolutely important. Vital even. But don’t forget about research.
Because it is only through research that we can truly make progress.
Lea Milligan is CEO of MQ Mental Health Research, a charity that funds world-class research and innovation to create better mental health care.