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Perspective on the Rx Pipeline: A Summary of Impactful Changes

July 10, 2018

EnvisionRx continuously monitors the drug pipeline. Our team evaluates changing treatment options in order to educate and inform our clients on the most impactful medications in development, new drugs recently approved, generics being launched and current medications receiving new indications.

Clinical Pipeline

Within the extensive pipeline of drugs preparing for market launch, four notable drugs plan sponsors should be aware of are:

  • Binimetinib/encorafenib* a combination treatment for BRAF-mutant advanced, unresectable or metastatic melanoma had a 39% reduced risk of death, improved median progression-free survival (PFS) rate and patients were able to tolerate it for longer than monotherapies of vemurafenib or encorafenib in clinical trials. Binimetinib/encorafenib represents the longest PFS rate compared to other commercially available products, however, it is dosed twice daily as compared to other agents at once daily dosing.

  • Elagolix for the management of endometriosis with associated pain and uterine fibroids was found to have a significantly higher clinical response rate for dysmenorrhea and non-menstrual pelvic pain compared to placebo in clinical trials. Patients also needed less rescue pain medication. In another study, combined with low-dose hormone therapy, patients saw a reduction in heavy menstrual bleeding. Elagolix may offer a second-line treatment option for patients that have been intolerant to other therapies and could delay the need for surgical intervention.

  • Fremanezumab if approved, will be the second drug in a new class of medications known as calcitonin gene-related peptide (CGRP) antagonists used for the prevention of migraines. Both fremanezumab and the previously approved erenumab are monoclonal antibodies, but fremanezumab targets the CGRP ligand and not the actual receptor like erenumab. With the potential of a quarterly regimen, fremanezumab may offer more convenient dosing than erenumab, which has a monthly schedule. Currently, there is no long-term safety data, but this does show promise as a significant advancement in migraine therapy in almost 10 years.

  • Lanadelumab if approved, will be the first kallikrein inhibitor available for the prevention of hereditary angioedema attacks. Clinical trials with multiple dosing found between 44.8% and 76.9% of patients taking lanadelumab were attack-free versus 2.7% with placebo. Lanadelumab also has less frequent dosing than other competitor products.

*During publication, the FDA approved these drugs as Mektovi® and Braftovi

Drug Approvals

Six significant specialty drugs recently approved by the Food and Drug Administration (FDA) include:

  • Aimovig (erenumab-aooe) a migraine prophylaxis

  • Andexxa® (andexanet alfa) for reversal of Factor Xa inhibitors

  • Crysvita® (burosumab-twza) for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients over one year of age

  • Epidiolex® (cannabidiol) adjunctive treatment of seizures associated with Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome

  • Lucemyra (lofexide hydrochloride) for the treatment of opioid use disorder

  • Retacrit (epoetin alfa-epbx) for the treatment of anemia

New Indications

Drugs that have gained FDA approval for the treatment of additional diseases and/or conditions and have potential to be impactful include:

  • Gilenya (fingolimod) was developed to treat patients with relapsing forms of multiple sclerosis (MS) and in May, was approved as the first treatment for pediatric MS patients age 10 and older.

  • Opdivo® (nivolumab) is used for the treatment of a variety of cancers and in April, was approved in combination with Yervoy® to treat patients with intermediate and poor-risk advanced renal cell carcinoma.

  • Truvada® (emtracitabine and tenofovir) was approved for the treatment of HIV-1 in August 2004. In May, the FDA expanded the indication for reducing the risk of acquiring HIV-1 in adolescents.

  • Xeljanz® (tofacitinib) is used to treat adults with rheumatoid and psoriatic arthritis. In May, it was also approved for the treatment of moderately to severely active ulcerative colitis.

Generic Approvals

There are several new generic product offerings to the marketplace, including a drug for female infertility, a dry eye medication (Restasis®), a treatment for HIV and two acne medications. A glimpse into the potential generic launches is included in the full report.

Download the full report for more information, including important safety updates and drug shortages and discontinuations.