Jazz Pharma’s sleep disorder treatment gets US FDA nod

This Oct. 14, 2015 file photo shows the Food & Drug Administration campus in Silver Spring, Maryland. (AP Photo/File)
Updated 21 March 2019
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Jazz Pharma’s sleep disorder treatment gets US FDA nod

  • The drug, solriamfetol, will treat excessive sleepiness in adult patients with narcolepsy or obstructive sleep apnea
  • The patent of Jazz's narcolepsy drug, Xyrem, were declared invalid by a US appeals court in July
The US Food and Drug Administration approved Jazz Pharmaceuticals Plc’s treatment for patients with a form of sleep disorder, the company said on Wednesday.
The drug, solriamfetol, will treat excessive sleepiness in adult patients with narcolepsy or obstructive sleep apnea (OSA).
Solriamfetol is expected to be commercially available in the United States following the final scheduling decision by the Drug Enforcement Administration (DEA), Jazz said in a statement.
The approval comes as Jazz is trying to reduce its reliance on its blockbuster narcolepsy drug, Xyrem, whose patents were declared invalid by a US appeals court in July.
Xyrem is an approved treatment for excessive daytime sleepiness and cataplexy in patients with narcolepsy. It brought in sales of $1.4 billion in 2018 and accounted for about 70 percent of company’s revenue.
“Jazz is trying to reduce its reliance on Xyrem, and solriamfetol will be one of the drugs it plans to launch to do that,” Mizuho Securities USA analyst Irina Koffler said ahead of the agency’s decision.
“Solriamfetol is expected to be an important driver of both diversification and growth,” RBC Capital Markets analyst Randall Stanicky said in a note ahead of the approval.
Solriamfetol is expected to bring in revenue of $314 million by 2024, Stanicky said.
Narcolepsy is a chronic sleep disorder with overwhelming daytime drowsiness and sudden attacks of sleep, while obstructive sleep apnea is a potentially serious sleep disorder that can cause breathing to repeatedly stop and start.
“Narcolepsy is very disabling to people as they often get diagnosed young and stop their education and drop out of high school or college,” Koffler said.
“Sleep apnea is a different problem in the sense that a lot of people don’t know they have it, have trouble breathing at night and they even fall asleep during the day.”


Blood donation in the Middle East: The gift of life that is easy to give

Updated 14 June 2019
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Blood donation in the Middle East: The gift of life that is easy to give

  • World Blood Donor Day observed on June 14 to raise awareness of the life-saving importance of blood donation
  • Regular, voluntary donors are vital worldwide for adequate supply of safe blood and blood products

DUBAI: Blood donations in the Middle East have been described as “the gift of life” as the region struggles to cope with the demands posed by conflicts, humanitarian emergencies and the medical needs of a growing population.

International health experts have called on regular donors to step forward to mark World Blood Donor Day on June 14.

This year’s campaign focuses on blood donation and universal access to safe blood transfusion, and according to the World Health Organization (WHO), more donors are needed “to step forward to give the gift of life.”

Those who benefit most from blood donations include people suffering from thalassaemia, a blood disorder that affects hemoglobin and the red blood cell count, as well as victims of road accidents, cancer patients and sickle-cell disease patients.

Experts say while the Gulf Cooperation Council (GCC) countries have launched numerous initiatives to raise awareness of the lifesaving importance of blood donation, there is an increasing need across a wider region for regular donors.

“Many countries in the region face challenges in making sufficient blood available while also ensuring its quality and safety, especially during humanitarian emergencies and conflicts,” Dr. Ahmed Al-Mandhari, WHO regional director for the Eastern Mediterranean, told Arab News.

The GCC countries say they collect in total more than 10 whole blood donations per 1,000 population per year, or about 1 percent, Al-Mandhari said.

According to WHO, blood donations by 1 to 3 percent of the population are sufficient to meet a country’s needs. Even so, achieving self-sufficiency is a daunting challenge for many countries.

Al-Mandhari said that more than 90 percent of the blood is collected from voluntary, unpaid donors, aged from 18 to 44, with an increasing proportion of repeat donors. What is more, blood demand is unpredictable and even differs with each blood type. “For example O- blood can be given to patients with all blood types. But AB+ can only be given to patients with AB+,” he said.

Then there is the issue of short shelf life.

“To be ready to help patients in all hospitals, countries aim to stock usually six days’ worth of each blood type at all times,” Al-Mandhari said. “Since blood has a short shelf life — a 42-day window — and cannot be stockpiled, blood banks are forced to depend on donors to help maintain stocks.”

WHO’s most recent report on blood safety and availability points to “gaps in the key elements of national blood systems” in the Middle East.

A Saudi donor flashes the v-sign for victory as he gives blood in Jeddah. The Kingdom has one of the highest rates of repeat donors in the region. (AFP )

While GCC countries have taken steps to keep stocks at optimum levels, other countries in the Middle East are lagging behind international standards. The WHO report shows wide variations in annual blood-donation rates among countries, ranging from 0.7 per 1,000 population in Yemen to 29 per 1,000 population in Lebanon.

Al-Mandhari laid out the solution in a few easy steps: “Governments need to provide adequate resources, and put in place systems and infrastructure to increase the collection of blood from voluntary, regular unpaid blood donors, provide quality donor care, promote and implement appropriate clinical use of blood; and set up systems for oversight and surveillance across the blood-transfusion supply chain.”

On the positive side, Saudi Arabia recorded a rate of 13.8 per 1,000 population, with a healthy spread across all age groups. The country also has one of the highest rates of repeat donors (91 percent) in the region. According to the WHO report, the proportion of repeat, voluntary, non-remunerated blood donation in the Kingdom is 65.3 percent, which “will keep the prevalence of transfusion-transmissible infections among blood donors at much lower levels than in the general population.”

In recent years, Saudi health officials have introduced a number of measures to ensure adequate stocks in blood banks, including those run by the Ministry of Health and dedicated centers. These include a large facility at King Fahad Medical City (KFMC) and the country’s Central Blood Bank.

In the Kingdom, to be eligible for blood donation, donors must be aged over 17, weigh more than 50 kg, and have passed a brief medical examination. The health ministry recently launched Wateen, an app designed to ease blood-donation procedures and help ensure facilities across the Kingdom have adequate quantities of blood by 2020.

KFMC officials say that every day at least 2,000 units of blood components are needed to sustain a minimum supply for patients at the facility and other governmental and non-governmental hospitals in Riyadh. Donated blood components are essential for the management of cases involving cancer, sickle-cell disease, organ transplant, surgery, childbirth and trauma, to name just a few.

The situation is not very different in the other GCC countries, which also need more donors.

In the UAE, Dubai Blood Donation Center, which accounts for roughly half of the total blood collected in the emirates, frequently highlights the urgent need for donors. In 2018 alone, it ran 635 blood-donation campaigns, which resulted in 63,735 donors and a collection of 50,456 blood units.

While all blood types are needed, negative blood types are in greater demand due to their rarity. “There is a continuous demand for all blood types as blood lasts for only 42 days. So donors are always needed to come forward to replenish these stocks,” Dr. Mai Raouf, director of Dubai Blood Donation Center, said.

“People can donate blood every eight weeks, with each donation potentially saving up to three lives,” she told Arab News. 

Given that transfusion of blood and blood products save millions of lives every year, and the fact that “regular donors are the safest group of donors,” the importance of encouraging people to return to donate blood, rather than be one-time donors, can hardly be overemphasized, experts say.

“Without a system based on voluntary, unpaid blood donation, particularly regular voluntary donation, no country can provide sufficient blood for all patients who require transfusion,” Al-Mandhari said.

“WHO is calling on all countries in the region to celebrate and thank individuals who donate blood — and to encourage those who have not yet donated blood to start donating,” he said.