Prescribe Right Pharmaceutical Pipeline Update for October 11

​Approvals

• The FDA approved Almirall’s and Paratek Pharmaceuticals’ sarecycline (Seysara) on 10/2/18, ahead of the PDUFA Date, for the treatment of patients aged 9 years and older with inflammatory lesions associated with non-nodular moderate to severe acne vulgaris. No Priority Designations.
• The FDA approved Paratek Pharmaceuticals' omadacycline (Nuzyra) on 10/2/18, ahead of its PDUFA Date, for the treatment of adults with community-acquired bacterial pneumonia or acute skin and skin structure infection. Omadacycline is a once-daily IV and oral antibiotic. Pricing is expected to be similar to linezolid (Zyvox). The drug had Fast Track and Qualified Infectious Disease Product Priority Designations.
• The FDA has approved Ionis Pharmaceuticals’ inotersen (Tegsedi) on 10/5/18, for the treatment of hereditary transthyretin-mediated amyloidosis polyneuropathy in adults. Inotersen is a once-weekly subcutaneous injection. Inotersen will be available through a REMS program with distribution through Accredo specialty pharmacy, a subsidiary of Express Scripts. The drug has a black box warning for thrombocytopenia and glomerulonephritis. Inotersen will be priced at $450,000. ICER concluded the drug would need to be priced at $96,103 to be cost-effective. The drug has Fast Track and Orphan Drug Priority Designations.

The FDA rejected ferric maltol during Shield’s first submission, but the company has resubmitted an NDA, which was accepted by the FDA this month.

Tocagen announced that in a Phase I trial, patients treated with Toca 511 and Toca FC had an overall survival rate of 35% at 2 years and 26% at 3 years. Toca 511 and Toca FC have FDA Breakthrough Therapy Priority Designations. The EMA designated Toca 511 & Toca FC for orphan status.

Viking announced top line results from a Phase II trial in patients recovering from hip fracture surgery. The highest dose of VK5211 (2 mg) increased lean body mass 9.1%, decreased fat mass 6.2% and increased 6-minute walking distance by 22 meters compared to placebo. At 24 weeks, increased lean body mass was maintained, but was similar to placebo.

In a crossover trial, Jazz’s solriamfetol, PDUFA Date of Dec 20, 2018, was found to have abuse potential that was similar to or lower than phentermine. The drug has an Orphan Drug Priority Designation.

ObsEva announced that in a Phase III trial, nolasiban improved the live birth rate 25% to 35% compared to placebo. ObsEva plans to file an MAA in the EU in 2019.

Arena announced that in a Phase II trial in patients with pulmonary arterial hypertension (PAH), ralinepag demonstrated a 29.8% improvement in pulmonary vascular resistance compared to placebo. Arena announced interim data from their open label extension of their Phase II PAH trial, where treatment with ralinepag continued to demonstrate an improvement in pulmonary vascular resistance and 6-minute walk distance in both patients that continued on ralinepag (1.8 years) and patients that crossed over from placebo (1.4 years). The drug has an Orphan Drug Priority Designation. Current trial data display no significant safety concerns; no independent safety data are yet available. The most common ADR reported for ralinepag in a Phase II trial were headache and nausea.

Omeros announced that in a Phase II trial, data suggested OMS721 improved kidney function and a reduced corticosteroid dose in IgAN, membranous nephropathy, lupus nephritis, and complement component 3 (C3) glomerulopathy. The drug has Fast Track, Breakthrough Therapy and Orphan Drug Priority Designations. Current trial data display no significant safety concerns; no independent safety data are yet available. The most common ADR reported for OMS721 in a Phase II trial were fatigue and anemia. Omeros has anongoing Phase III trial evaluating OMS721 in the treatment of Immunoglobulin A (IgA) Nephropathy and as a treatment for glomerulonephropathies and hematopoietic stem cell transplant-associated thrombotic microangiopathy.

Roche announced that in a Phase III trial, a single dose of baloxavir marboxil reduced the time to alleviation of symptoms by 29.1 hours compared to placebo and was similar to 5 days of oseltamivir in influenza patients at high risk for complications. Baloxavir marboxil also reduced viral shedding more rapidly than oseltamivir or placebo. PDUFA Date: 12/24/2018. In the safety study that was part of the Phase III trial, nausea was less common with a single dose of baloxavir marboxil than 5 days of oseltamivir.

​Sarepta announced interim results from the first 4 patients in a 27 patient, Phase I/II trial, where microdystrophin gene therapy increased micro-dystrophin levels to 74% of normal dystrophin levels, though it is not currently known if microdystrophin has the same functional benefit as normal dystrophin.

Ibudilast was granted orphan status by the FDA.

The FDA granted a priority review for Karyopharm Therapeutics’ selinexor and assigned a PDUFA date of April 6, 2019.

Stay current with drugs in the late stages of development with the Prescribe Right Pharmaceutical Pipeline Tracker.