Pricing Reimbursment and Market Acces Newsletter #21

NICE terminates appraisal of afatinib dimaleate

24 May 2017 — NICE was to assess afatinib dimaleate for treatment of patients with advanced squamous non-small-cell lung cancer after platinum-based chemotherapy.

NICE is unable to make a recommendation about the use in the NHS of afatinib for treating locally advanced or metastatic squamous non-small-cell lung cancer after platinum-based chemotherapy because no evidence submission was received from Boehringer Ingelheim.

NICE will review this decision if the company decides to make a submission.

Full article here.

FDA approves Merck’s Keytruda (pembrolizumab) for adult and pediatric patients with unresectable or metastatic, microsatellite instability-high or mismatch repair deficient cancer

23 May 2017 — Keytruda now approved for patients with MSI-H or mismatch repair deficient solid tumours that have progressed following prior treatment and who have no satisfactory alternative treatment options, which includes MSI-H or mismatch repair deficient colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan.

Merck today announced that the U.S. FDA has approved a new indication for Keytruda (pembrolizumab), the company’s anti-PD-1 therapy. Keytruda is now indicated for the treatment of adult and paediatric patients with unresectable or metastatic, microsatellite instability-high or mismatch repair deficient:

  • solid tumours that have progressed following prior treatment and who have no satisfactory alternative treatment options, or
  • colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan.

This indication is approved under accelerated approval based on tumour response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Full article here.

CADTH recommends reimbursement of Gilead’s Odefsey

25 May 2017 — The CADTH Canadian Drug Expert Committee has recommended that emtricitabine/rilpivirine/tenofovir alafenamide be reimbursed as a complete regimen for the treatment of adults infected with human immunodeficiency virus type 1 who have no known mutations associated with resistance.

The cost of Odefsey should not exceed the cost of FTC/RPV/tenofovir disoproxil fumarate (TDF) or the individual components of Odefsey used in combination.

Full article here.

Transparency Commission publishes its review of Keytruda’s SMR & ASMR

22 May 2017 — The Transparency Commission has reviewed its SMR & ASMR ratings of pembrolizumab for malignant melanoma.

The available data based on an update of the follow-up of the two studies already evaluated by the Commission (KEYNOTE 001 and KEYNOTE 006) are not likely to modify the assessment of improvement of the minor medical rendering service (ASMR IV) Attributed to Keytruda by the Commission in its opinion of 16 March 2016.

Full article here.

CalciMedica receives fast-track designation for CM4620, a novel CRAC channel inhibitor to treat acute pancreatitis

24 May 2017 — Patient studies expected to begin in 2018.

CalciMedica announced today that it received fast-track designation from the U.S. FDA for its lead clinical compound, CM4620, a novel, calcium release-activated calcium (CRAC) channel inhibitor, for the treatment of acute pancreatitis.

Currently, there are no therapeutic agents approved to treat acute pancreatitis, an inflammatory condition that is the leading cause of gastrointestinal hospitalisations and healthcare cost burden in the US.

Full article here.

Health Canada grants approval for Ocaliva (obeticholic acid) for the treatment of patients with primary biliary cholangitis

25 May 2017 — Ocaliva represents the first new treatment option in over 20 years for primary biliary cholangitis, an autoimmune liver disease that is the leading reason for liver transplantation in women in Canada.

Intercept Pharmaceuticals today announced that Health Canada has granted a conditional approval for Ocaliva (obeticholic acid) for the treatment of primary biliary cholangitis, when used in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.

Full article here.

Hepatera: eligibility to PRIME scheme granted to Myrcludex B by the European Medicines Agency

25 May 2017 — MYR and its development partner Hepatera today announced that the EMA granted “PRIME eligibility” for Myrcudex B, a first in class entry inhibitor for Hepatitis Delta (D) virus.

Myrcludex B is a first in class entry inhibitor for treatment of chronic hepatitis B and D. The drug has shown promising efficacy in Phase 2a trials. Recruitment for Phase 2b program in HDV is completed.

Full article here.

Novartis receives FDA approval for expanded use of Zykadia in first-line ALK-positive metastatic non-small cell lung cancer

26 May 2017 — In ALK-positive metastatic NSCLC patients, Zykadia median progression-free survival was 16.6 months, compared to 8.1 months with chemotherapy.

Novartis today announced the US FDA approved the expanded use of Zykadia (ceritinib) to include the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumours are anaplastic lymphoma kinase (ALK)-positive, as detected by an FDA-approved test.

Zykadia first received accelerated approval in 2014 for patients with ALK-positive metastatic NSCLC who progressed on or are intolerant to crizotinib. In January 2017, the FDA granted Zykadia breakthrough therapy designation for the first-line treatment of patients with ALK-positive metastatic NSCLC with metastases to the brain, and priority review for first-line ALK-positive metastatic NSCLC.

Full article here.

UnitedHealth and Merck strike a deal to explore linking payments to drug performance

25 May 2017 — UnitedHealth Group’s Optum unit has signed a multiyear agreement with Merck to develop a better way to reimburse drugmakers based on how well their drugs improve a patient’s health.

“The aim is to identify opportunities where we can align value and payment,” on high-cost treatments, said Curt Medeiros, president of Optum Life Sciences. “There’s a lot of focus on high-cost specialty areas like oncology, but also in chronic conditions that affect a broad set of the population like diabetes.”

UnitedHealth and other insurers have been pushing hospitals and doctors toward new value-based reimbursement contracts for more than five years. In the last 18 months, the health insurance industry has begun to focus more on new payment deals with drugmakers to enter outcomes-based risk sharing agreements, or OBRSAs.

Full article here

Sunovion receives complete response letter from FDA for SUN-101/eFlow (glycopyrrolate) new drug application for chronic obstructive pulmonary disease

26 May 2017 — Sunovion Pharmaceuticals today announced that the U.S. FDA issued a complete response letter for the new drug application for SUN-101/eFlow (glycopyrrolate) for the long-term, maintenance treatment of airflow obstruction in patients with chronic obstructive pulmonary disease, including chronic bronchitis and/or emphysema.

The CRL does not require Sunovion to conduct any additional clinical studies for the approval of SUN-101/eFlow.

SUN-101 (glycopyrrolate) is a long-acting muscarinic antagonist bronchodilator delivered via the proprietary investigational eFlow closed system nebuliser. SUN-101/eFlow is currently in development as a nebulised treatment for patients with moderate-to-very-severe chronic obstructive pulmonary disease (COPD). The investigational combined product, consisting of SUN-101 and the investigational eFlow closed system nebuliser, which has been optimised for SUN-101 delivery, has not been approved by the FDA for the treatment of COPD.

Full article here.

Kite receives U.S. Food and Drug Administration priority review for axicabtagene ciloleucel

26 May 2017 — Biologics license application submission based on the primary analysis of the ZUMA-1 Phase 2 trial.

Kite Pharma today announced that the U.S. FDA has accepted for priority review the biologics license Application for axicabtagene ciloleucel.

The submission follows positive data demonstrated with a single infusion of axicabtagene ciloleucel in the ZUMA-1 Phase 2 trial in patients with refractory aggressive non-Hodgkin lymphoma.

The FDA has set a Prescription Drug User Fee Act target action date of 29 November 2017.

Full article here.

FDA grants priority review to Merck’s supplemental biologics license application for Keytruda (pembrolizumab) for treatment of recurrent or advanced gastric or gastro-oesophageal junction adenocarcinoma

23 May 2017 — Data supporting application to be presented at 2017 ASCO Annual Meeting.

Merck announced today that the U.S. FDA has accepted for review a supplemental biologics license application for Keytruda (pembrolizumab), the company’s anti-PD-1 therapy, seeking approval for treatment of patients with recurrent or advanced gastric or gastroesophageal junction adenocarcinoma who have already received two or more lines of chemotherapy.

The FDA granted priority review with a PDUFA, or target action, date of 22 September 2017.

Full article here.

Japan to bring forward target for increased generic drug use

22 May 2017 — Japan will bring forward a target to boost the use of generic drugs by six months to September 2020, two government sources with direct knowledge of the matter said on Tuesday.

The rapidly ageing nation is aiming to lift the use of generics to more than 80% from around 56% currently — a move that would save the government hundreds of billions of yen a year, the sources said.

The government will set the target early next month as part of its annual key economic and fiscal policy guidelines, the sources added, declining to be identified as they were not authorised to speak to the media.

Full article here.

Plazomicin granted FDA breakthrough therapy designation

23 May 2017 — Breakthrough therapy designation supports the potential of plazomicin as a substantial improvement over existing therapies

Achaogen today announced that the U.S. FDA granted breakthrough therapy designation for plazomicin, Achaogen’s lead product candidate being developed for the treatment of serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae.

The Phase 3 CARE trial data provided clinical evidence to support the breakthrough Therapy Designation for plazomicin for the treatment of bloodstream infections caused by certain Enterobacteriaceae in patients who have limited or no alternative treatment options.

Full article here.

Ultragenyx announces recombinant human beta-glucuronidase biologics license application and marketing authorisation application filed and accepted for review; FDA grants priority review status

23 May 2017 — Ultragenyx today announced that a biologics license application submitted to the U.S. FDA and a marketing authorisation application submitted to the EMA, for recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of mucopolysaccharidosis VII (MPS VII, Sly syndrome) were accepted for review.

The Prescription Drug User Fee Act goal date for a decision is 16 November 2017 and an opinion from the CHMP is expected in the first half of 2018. rhGUS is an enzyme replacement therapy for the treatment of MPS VII.

The FDA granted rhGUS priority review status, which is available for drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists. rhGUS was previously granted orphan drug designation by the FDA.

Full article here.

European Commission grants marketing authorisation for Zebinix (eslicarbazepine acetate) as once daily monotherapy in adults with newly diagnosed partial-onset epilepsy

23 May 2017 — Bial and Eisai today announce that the EMA has approved Zebinix (eslicarbazepine acetate) for use as a once-daily monotherapy to treat adults with newly-diagnosed partial-onset epilepsy.

Eslicarbazepine acetate is already indicated in Europe as adjunctive therapy in adults, adolescents and children aged above six years, with partial-onset seizures with or without secondary generalisation.

Full article here.

U.S. Food and Drug Administration accepts for priority review Bristol-Myers Squibb’s application for Opdivo (nivolumab) in previously treated hepatocellular carcinoma

24 May 2017 — Application is based on results from the Phase 1/2 CheckMate-040 trial.

Bristol-Myers Squibb Company today announced that the U.S. FDA accepted a supplemental biologics license application that seeks to extend the use of Opdivo (nivolumab) to patients with hepatocellular carcinoma (HCC) after prior sorafenib therapy.

The FDA granted the application priority review and previously granted Opdivo orphan-drug designation for the treatment of HCC.

The FDA action date is 24 September 2017.

Full article here.