Module 2: Discussion Group A by Supriya Munshaw https://padlet.com/smunshaw/4gsnv840v0aa Find a recent news story from the last 6 months that showcases a R&D strategy used by a pharmaceutical company (see Changing R&D models article). Comment on why they may have chosen to go that route and provide your thoughts; for example, what can you determine based on their pipelines? en-us 2018-02-06 16:12:07 UTC 2026-01-07 08:04:53 UTC hello@padlet.com Sarepta Announces Plans to File New Drug Application for Golodirsen - entry by Danny Goldberg https://padlet.com/smunshaw/4gsnv840v0aa/wish/246336547 On March 12, Sarepta Therapeutics announced their intent to submit a new drug application (NDA) for accelerated approval of their drug golodirsen to the FDA. If approved, this would be their second commercialized product. Looking across Sarepta's pipeline, they are focused on treating children with Duchenne Muscular Dystrophy (DMD) amenable to exon skipping of the DMD gene. DMD is a disease characterized by muscle degeneration and weakness; there are 9 types of muscular dystrophy. The exon skipping works to restore dystrophin, which is the protein DMD patients lack that keeps muscles together. 

In assessing this development, Sarepta's decision to file an NDA for their next DMD drug in the pipeline suggests an accelerated growth strategy, as golodirsen wasn't expected to be commercialized until 2020. Sarepta is likely pursuing this strategy for 2 reasons: 1) to take a clear lead in the DMD space in terms of market share for therapies, and 2) to execute on a their vision to be "one of the most important genetic medicine companies in the next five years," according to CEO Douglas Ingram. 

There is a need to go at a feverish pace if Sarepta wants to achieve this vision. According to the WSJ, competitors Novartis and Spark Therapeutics have taken a lead in the precision genetic medicine field. Following the JP Morgan conference, the largest annual health care conference, the discussion and exploration of gene therapy has gained significant public traction. Sarepta likely felt increasing pressure from shareholders after this conference at the beginning of the year to make a bold splash in a competitive gene therapy market. Getting this product commercialized quickly sets the company up for financial success over time and makes a statement that Sarepta is a name to compete with in genetic medicine development.

]]> 2018-03-27 02:17:09 UTC https://padlet.com/smunshaw/4gsnv840v0aa/wish/246336547 On Feb 8, 2018, Gilead got an approval from FDA for their new drug launch “HIV triplet” drug (bictegravir/FTC/TAF, Gilead Sciences) by Eyerusalem Yemane https://padlet.com/smunshaw/4gsnv840v0aa/wish/246595517 On Feb 8, 2018, Gilead got an approval from FDA for their new drug launch “HIV triplet” drug (bictegravir/FTC/TAF, Gilead Sciences).  It’s Known as BIC/F/TAF in the trade name, combining the novel bictegravir with the backbone combination of emtricitabine/tenofovir alafenamide into a single daily pill. This will replace a regimen that once required a multitude of pills to keep the virus under control. In mid-2017, Gilead said that four phase 3 studies evaluating a fixed-dose combination of bictegravir (BIC), a novel investigational integrase strand transfer inhibitor (INSTI), and emtricitabine/tenofovir alafenamide (FTC/TAF) to treat HIV-1 met their primary objectives of non-inferiority to the existing HIV drugs.  

The company is pushing for this drug as the existing HIV drugs will hit patient expiry soon.  This means Gilead’s will be trying to convert patients to the more convenient regimen which will give them power in the market.  As being a sole maker for this single drug, the company can set a price to benefit the com.  

“All indications are that Gilead’s new HIV triplet can reach $5 billion in sales by 2022, based on the strength of their HIV franchise and the company’s willingness to spend resources to remain the market leader” according to the company CEO.

The second reason why the company spend so much for developing this drug is that the company realized that their sales in hepatitis C market decreases.  This will be an ideal way to stay in the market and make money at the same time. 

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