Five-month old baby first to receive world’s most expensive gene therapy drug on the NHS
The UK’s National Health Service (NHS) has treated Arthur Morgan, a five-month old baby diagnosed with Spinal Muscular Atrophy (SMA), with Novartis’ SMA gene therapy drug Zolgensma.
SMA is a rare, inherited, condition that affects motor neurons in the spinal cord, and affects approximately 1 in 10,000 live births. If left untreated, children rarely live beyond the age of two.
Until two years ago, there were no treatment options available for children with SMA, but now gene therapy can potentially give babies the ability to sit, crawl and walk.
Zolgensma has proved to show a “long-term favorable safety profile” in children aged up to six years with SMA Type 1, according to findings published in JAMA Neurology. The gene therapy has not been designed to cure SMA, but rather halt the progression of the disease.
Olga Santiago, Chief Medical Officer at AveXis, a Novartis company, said: “Patients who received Zolgensma soon after birth before the onset of symptoms are achieving age-appropriate motor milestone development.”
Accordding to Santiago, the data from the clinical trial examining the long-term efficacy of the drug “is demonstrating the truly transformational impact a one-time dose of gene therapy can have, and further underscores the importance of newborn screening and early intervention to alter the course of the disease”.
Novartis’ Zolgensma has a listed price point of £1.8m (US$2.4m) per dose, and was made available to the NHS after the health service struck a deal with the drug manufacturers in March, The Guardian reported.
Dad to baby Arthur Morgan, said: “[Zolgensma] is the best possible treatment and will give him the best life he can have, and we are so grateful that the NHS is here for him.”
Simon Stevens, Chief Executive of the NHS, said: “It is fantastic news that this revolutionary treatment is now available for babies and children like Arthur on the NHS.
“The NHS Long Term Plan committed to securing cutting edge treatments for patients at a price that is fair to taxpayers.
“Zolgensma is the latest example of the life-changing therapies that the NHS is now routinely using to transform the lives of patients and their families.”
Congestion at China’s Yantian port worsens building to unprecedented supply chain disruption
South China’s port congestion has worsened with shipping containers at Yantian now spilling over into the neighboring ports, Shekou and Nansha, due to the Covid-19 outbreak of the Delta variant in the Yantian port area.
The restriction measures that are in place to combat the coronavirus outbreak combined with the rapid reopening of the global economy and a surge in transportation costs of pharmaceuticals has caused additional delays to shipping operations at Yantian port.
Major shipping companies for pharma products such as Maersk have warned clients of the worsening congestion and top international container lines have said they would be skipping pit calls to Yantian to ease the pressure, Reuters reported.
Vincent Clerc, Chief Commerical Officer at Maersk, told Seatrade Maritime News: “I would say this for us is a much bigger disruption than the Ever Given getting stuck in the Suez Canal for some days because of the duration and the importance of Yantian as a gateway.”
The blockage of ships at Suez Canal lasted for six days, while the unceasing situation in Yantian has continued for several weeks already. A resolution for the unprecedented disruption has yet to be announced.
Supply chain disruptions like these can have major affects on the quality of healthcare provisions and can negatively impact hospital performance.
Before the situation looks to improve, ports including Yantian, Shekou, Chiwan and Nansha, have barred any further shipping vessels from entering their ports without advance reservations and warn they will only accept bookings for export-bound containers within three to seven days prior to the arrival of vessels, The Guardian emphasized.
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