Vertex Pharmaceuticals Sets Sight on Expanding Portfolio with Alyftrek Approval, Pain Treatment Progress, and Diabetes Trials

On Sunday, Vertex Pharmaceuticals Incorporated VRTX announced multiple program updates, including:

• In December, the FDA approved Vertex's Alyftrek (vanzacaftor / tezacaftor / deutivacaftor), a once-daily triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator for cystic fibrosis (CF) in people 6 years and older who have at least one F508del mutation or another mutation in the CFTR gene that is responsive to ALYFTREK. Vertex raised its CF patient estimates across major markets to 94,000, while reimbursement arrangements are expected to broaden treatment access for 10,000 eligible individuals.

Also Read: Vertex Pharmaceuticals: Alyftrek Is An ‘Accretive Opportunity' On Top- And Bottom-Lines, Says Bullish Analyst

• In December, Vertex's CASGEVY gained regulatory approval in the UAE for sickle cell disease and transfusion-dependent beta-thalassemia.
• Suzetrigine for moderate-to-severe acute pain awaits an FDA decision by January 30, 2025, under Priority Review. This non-opioid candidate aligns with the newly effective NOPAIN Act, potentially improving pain management alternatives and ensuring Medicare support.
• In type 1 diabetes, zimislecel entered Phase 3 trials targeting 125,000 patients. Complementary therapies, including encapsulated islet cells in VX-264, are advancing, with pivotal data expected in 2025.

RBC Capital Markets says Vertex management is optimistic about the recently approved Alyftrek. They predict that most of the 6,000 patients who stopped using other CFTR modulators and many currently on existing treatments will switch to this next-gen therapy.

RBC analyst Brian Abrahams writes that in acute pain, Suzetrigine pricing may be higher than anticipated, as the company emphasized that it doesn't want to risk the long-term value of its pain portfolio or the success in chronic pain just for quick approval of its first drug. The analyst also noted that formulary placement and payer discussions will affect the initial launch.

RBC Capital maintains a Neutral rating on Vertex.

Goldman Sachs analyst Salveen Richter writes suzetrigine could become the first non-opioid treatment for acute pain approved in over 20 years.

It can generate billions in revenue from both acute and possibly chronic pain. If approved, it would give Vertex Pharmaceuticals a significant new revenue source beyond cystic fibrosis, especially as income from Casgevy remains low in the near term, helping to diversify its commercial portfolio.

Price Action: VRTX stock is down 0.46% at $411.47 at the last check on Tuesday.

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Image via Vertex Pharmaceuticals