FDA Panel Nod For GSK’s Shingles Vaccine, FOLD Beyond Essence, FENC Is Red Hot

Today’s Daily Dose brings you news about encouraging interim analysis of Alexion’s REGAIN study of Soliris; Fennec’s uplisting and preliminary results of SIOPEL 6; Amicus’ failed epidermolysis bullosa trial; Vanda’s disappointing atopic dermatitis trial results and FDA panel’s recommendation for approval of GlaxoSmithKline’s investigational shingles vaccine Shingrix.

Read on…

Alexion Pharmaceuticals Inc. (ALXN) has announced that interim analysis of its ongoing phase III open-label extension study of the pivotal, placebo-controlled REGAIN study of Soliris for the treatment of patients with refractory generalized myasthenia gravis show sustained treatment benefits.

The treatment benefits for patients treated with Soliris in REGAIN study through 26 weeks were maintained in the extension study across all four assessment scales for an additional 52 weeks (78 weeks in total), the company noted.

For patients who received placebo in REGAIN and then were treated with Soliris in the extension study, significant treatment benefits occurred within 1 to 4 weeks and were sustained through 52 weeks across all four assessment scales.

The assessment scales include MG-ADL – MG-Activities of Daily Living, a patient-reported assessment of functional ability to carry out daily activities; QMG – Quantitative MG, a clinical assessment of muscle strength by physicians; MGC – MG Composite, a patient- and physician-reported assessment of functional ability and signs and symptoms of MG and MG-QoL 15 – MG Quality of Life 15, a patient-reported assessment of MG-specific quality of life.

Soliris is already indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, and for the treatment of patients with atypical hemolytic uremic syndrome.

The drug generated total sales of $1.59 billion in the first half of 2017, and its sales are expected to range between $3.07 billion to $3.12 billion for the full year.

ALXN closed Wednesday’s trading at $146.49, up 1.68%.

Amicus Therapeutics Inc.’s (FOLD) phase III clinical study of novel topical wound-healing agent SD-101 in participants with epidermolysis bullosa, dubbed ESSENCE, did not meet the primary endpoints or secondary endpoints.

Epidermolysis bullosa is a rare, devastating genetic disorder that leads to severe skin blistering and open wounds often beginning at birth. There are no approved therapies to treat epidermolysis bullosa, which affects tens of thousands of children and adults…

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