Ricardo Batista and his wife call their baby daughter Eva a “warrior.”
Eva, born Aug. 11, 2019, was diagnosed with Type 1 Spinal Muscular Atrophy (SMA), at the age of only seven weeks.
SMA has been described as the number one genetic killer of children under the age of two, world-wide. The condition results in the death of motor neurons in the spine that control the muscles, producing muscle atrophy or wasting, loss of mobility, and in severe cases, the inability to swallow or breathe.
Some forms of SMA don’t develop until adulthood. Type 1 SMA, which attacks the youngest victims, is the most severe.
But there have been recent rays of hope.
In 2017, Health Canada approved the drug Spinraza, which increases the production of a protein that helps the motor neurons survive, and slows the onset of the disease.
And there is ZolgenSma.
ZolgenSma is a gene therapy, specifically for pediatric patients under the age of two – and it is being described as a cure.
ZolgenSma was developed through extensive research, largely funded by grants, and charitable donations from organizations like Cure SMA, Families of SMA and the French charity Genethon, at the University of Pennsylvania and Nationwide Children’s Hospital.
In 2018, AveXis – an offshoot of the Swiss company Novartis – purchased the worldwide rights to the new treatment. The idea was that AveXis would undertake further development, see the treatment through the approval process, and bring the new drug to market.
It did, but at a price. ZolgenSma, which received FDA approval in the U.S. earlier this year, is now being marketed at $2.1 million USD for the one-time treatment. It has not been approved in Canada.
For Eva’s family, the news has been both elating, and devastating. They see hope for a cure for their daughter, but the price is beyond their reach – beyond the reach of any family.
Eva is currently receiving Spinraza, and has already had three treatments, but Ricardo Batista has also launched a Go Fund Me page. The goal: to raise the $2.8 million (CAD) needed to cover the cost of the “most expensive drug in the world.”
On Nov. 9, the Portuguese Cultural Centre of Bradford (PCCB) turned their annual Sao Martinho Chestnut dinner and dance, featuring local band Sagres, into a fundraiser for Eva Batista.
Over 300 people attended the evening, raising a total of $5,610.
The PCCB has thanked everyone who donated, especially Jose and Rosa Melo who contributed $1,000; Frank and Jennifer of Cortale Haulage, Jon and Fina of Jon Marcus Homes, and John and Christina Alcaide whose donations totalled another $2,000; and nine-year-old Miguel Almeida, who donated the contents of his piggy bank.
Meanwhile, there are new concerns, that go beyond the price of the treatment.
ZolgenSma was approved with the warning that it may cause serious liver damage, but the FDA is now alleging that the information submitted by AveXis as part of the approval process was “manipulated.” While the drug has not been pulled from the market, the FDA has placed a partial hold on clinical trials while the data is being reviewed.
