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In the face of controversy, SMA's treatment drug Spinraza has welcomed a new competitor.
recently, a "domestic 700,000 yuan a drug in Australia only 41 Australian dollars" news rushed to the micro-blogging, and thus aroused renewed attention to the rare disease spinal muscular dystrophy (SMA) treatment.
is caught up in the controversy, developed by Biogen Idec Ltd and launched domestically in 2019, spinraza, which has drawn widespread criticism for making treatment too expensive for ordinary patients to bear.
Spinraza, once the only treatment for SMA, contributed $1.7 billion in sales to the drug in 2018 and revenue rose 22 percent to $2.1 billion in 2019 from 2018, including a 9 percent increase in the U.S. to $933 million and a 34 percent increase outside the U.S. to $1.2 billion.
As recently as August 7, 2020 local time in the United States, the drug welcomed a new competitor, Roche's Genentech company developed the oral drug Evrysdi to treat SMA was officially approved by the FDA, and it is also the first FDA-approved oral drug to treat SMA.
the drug has been approved for the treatment of infants and adults over 2 months of age with spinal muscular dystrophy (SMA).
it not only treats the most severely ill SMA infants and young children, but is also approved to treat adolescents and adults with relatively mild symptoms.
Evrysdi is a small molecule mRNA shear regulator for the SMN2 gene, developed by Gene Tek to PTC Therapeutics.
the SMN2 gene carried in the human body can also express SMN protein, but due to mRNA shearing error, resulting in normal SMN protein expression level is very low, can not make up for the SMN protein loss caused by the SMN1 gene mutation.
Evrysdi relieves symptoms in SMA patients by regulating the scissors of the SMN2 gene mRNA to increase the level of mRNA that expresses normal SMN proteins.
Evrysdi is a liquid-formulated drug that can be given orally at home or in liquid form through a feeding tube.
approval is based on two key clinical trials, FIREFISH and SUNFISH.
fireFISH study of infants aged 2-7 months, 41 percent of infants receiving treatment doses were able to sit for more than 5 seconds without assistance, a developmental milestone that infants without treatment could not reach.
, 81 percent of infants survive without permanent breathing after 23 months of treatment, a significant improvement over the natural development of the disease.
a total of 180 late-haired SMA patients aged 2-25 were included in the SUNFISH clinical trial.
using a performance evaluation test called MFM32, patients treated with Evrysdi had an average score of 1.36 after one year, while those in the placebo group had a 0.19 lower score.
side effects of Evrysdi include fever, diarrhea, rashes, mouth ulcers, joint pain (joint pain) and urinary tract infections.
in patients with infant-type SMA are similar to erythic SMA patients.
side effects of infant seizures include upper respiratory tract infections, pneumonia, constipation and vomiting.
patients should avoid taking Evrysdi in combination with a variety of drugs and toxin extrusion substrates, as Evrysdi may increase plasma concentrations in these drugs.
Evrysdi will be available in the US in two weeks.
according to Fierce Pharma media, Evrysdi is priced at $340,000 (about Rmb2.37m) a year, depending on the patient's weight, and for some younger patients it could be less than $100,000 (about Rmb700,000).
Spinraza, which is currently the focus of domestic attention, cost about $750,000 (about Rmb5.22m) in its first year of treatment in the US and dropped to $US375,000 (about Rmb2.61m) the next year and beyond. Novarro's Solgensma, known as
's most expensive drug of all time, is priced at $2.125m (about Rmb14.8m), but is a one-off treatment that requires only one administration and no subsequent annual fee.
According to Novarhua's recent results for the first half of 2020, Zolgensma achieved sales of $375 million in the first half of the year and $361 million last year.
statistics show that there are currently about 60,000 SMA patients worldwide, Spinraza covers about 11,110 people, so far spinraza has been approved for listing, it has brought in a cumulative sales of $5.7 billion, with the entry of Risdiplam, the current market structure will be broken.
Note: U.S. Dollar RMB Exchange Rate Conversion: US$1 x 6.9680 RMB References . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Evrysdi (risdiplam) for Treatment of Spinal Muscular Atrophy (SMA) in Adults and Children 2 Months and Older