Health Care

$89000 Drug: Marathon Charging $87800 More Than Rx's Price Overseas

$89000 Drug: Marathon Charging $87800 More Than Rx's Price Overseas

Corticosteroids are a staple for boys with Duchenne and until the approval of deflazacort, families would you use off label corticosteroids or buy deflazacort from overseas pharmacies.

DMD typically affects boys; however, girls can be mildly affected and be carriers of the gene, according to the Muscular Dystrophy Association. Unlike etelirsen, the drug will be approved for all DMD patients age 5 years and older. It is third drug in 6 months approved by the FDA for diseases included in MDA's program.

Individuals with DMD progressively lose the ability to perform activities independently and often require the use of a wheelchair by their early teens. Even though the drug has been available as a generic in other countries for quite some time, the federal designation also gives Marathon exclusive rights to market the drug in the US for seven years.

The company asked the FDA for approval of deflazacort as an orphan drug, which is a special pathway to approval that is meant to encourage more development of treatments for rare diseases.

O Panneerselvam at Madurai, villagers say no Jallikattu without permanent solution
A rekla (ox-cart) race, which was inaugurated by state Minister SP Velumani, was organised in Coimbatore on Sunday. Groups of agitators continued their protests at several locations in the state, raising the same demand.


Emflaza's approval also brings in a strong competitor for Sarepta, whose DMD drug Exondys 51 (eteplirsen) secured approval last September.

Even at a cost of $89,000, patient advocacy groups are enthusiastic about the FDA approval of deflazacort. The drug was never sold in the USA because companies didn't think it would be profitable enough, The Wall Street Journal reported, but some families have obtained the drug from online pharmacies overseas to help improve muscle strength for boys at least for a while. It improves muscle strength. Their ages ranged between 5 and 15 at the beginning of the trial. After 12 weeks, patients taking deflazacort saw improvements in a clinical assessment of muscle strength across several muscles compared with those taking a placebo. An overall stability in average muscle strength was maintained through the end of study at week 52 in the deflazacort-treated subjects.

While Emflaza has been available globally for the past few years as a DMD therapy, it was not available in the United States. The most common side effects include facial puffiness, weight gain, upper respiratory tract infection, cough, increased appetite and hair growth, and excessive fat around the stomach.

Other side effects that are less common include problems with endocrine function, increased susceptibility to infection, elevation in blood pressure, risk of gastrointestinal perforation, serious skin rashes, behavioral and mood changes, diminished bone density, and vision problems such as cataracts.


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