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2. Vertex Pharmaceuticals - Wikipedia

   en.wikipedia.org/wiki/Vertex_Pharmaceuticals

   In 2012 ivacaftor was designated as an orphan drug, identifying cystic fibrosis as affecting fewer than 200,000 people in the United States.On 31 January 2012, Vertex gained FDA approval [22] of the first drug, Kalydeco, [23] to treat the underlying cause of cystic fibrosis rather than the symptoms, in patients 6 years or older who have the G551D gene mutation.

3. Suzetrigine - Wikipedia

   en.wikipedia.org/wiki/Suzetrigine

   Vertex Pharmaceuticals announced in January 2024 that suzetrigine successfully met several endpoints in its phase 3 trials. The drug relieved moderate-to-severe post-surgical pain, comparably to an opioid–acetaminophen combination. [4] The company hopes that the drug, which operates on peripheral nerves, will avoid the addictive potential of ...

4. Exagamglogene autotemcel - Wikipedia

   en.wikipedia.org/wiki/Exagamglogene_autotemcel

   Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizing CRISPR/Cas9 gene editing technology to be approved by the US Food and Drug Administration (FDA). [13] The most common side effects include low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile ...

5. Vertex Pharma scientist talks about the long road to ... - AOL

   www.aol.com/lifestyle/vertex-pharma-scientist...

   Vertex Pharmaceuticals recently reported positive results for a non-opioid painkiller, one of several medications the Boston-based drugmaker has been developing for various forms of pain.

6. Vertex non-opioid painkiller meets main goal in late ... - AOL

   www.aol.com/news/vertex-non-opioid-pain-drug...

   (Reuters) -Vertex Pharmaceuticals' experimental drug reduced acute, post-surgical pain without the risk of addiction, a major milestone in the company's decades-long efforts to bring a non-opioid ...

7. Vertex's non-opioid drug reduces nerve pain in trial, shares ...

   www.aol.com/news/vertexs-non-opioid-drug-reduces...

   (Reuters) -Vertex Pharmaceuticals' non-opioid painkiller significantly decreased pain in patients with diabetes suffering from chronic nerve pain in a mid-stage trial, fueling efforts to develop a ...

8. List of drugs granted breakthrough therapy designation

   en.wikipedia.org/wiki/List_of_drugs_granted...

   Drug Manufacturer Indication Ibrutinib [1] Pharmacyclics: Waldenstrom’s macroglobulinemia: Palbociclib [1] Pfizer: ER-positive, HER2-negative breast cancer: Ranibizumab [1] Genentech: diabetic retinopathy: Ivacaftor [1] Vertex Pharmaceuticals: cystic fibrosis with a variety of CFTR mutations Aflibercept [1] Regeneron Pharmaceuticals: diabetic ...

9. Did Vertex Pharmaceuticals Just Invent the Next Ozempic? - AOL

   www.aol.com/did-vertex-pharmaceuticals-just...

   With Vertex Pharmaceuticals (NASDAQ: VRTX) announcing on June 21 that its therapy VX-880 helped nearly all of the patients with diabetes in a clinical trial to reduce or eliminate their need for ...