Pipeline News and Updates
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A light week, until Friday, December 21, then everything happened at once. Of the three drugs with December PDUFA dates, two were approved and one was delayed. The FDA also approved two drugs that did not have PDUFA dates to bring the 2018 total to 59.
The FDA approved prucalopride (Motegrity from Shire) on 12/14/18 for the treatment of adults with chronic idiopathic constipation. The FDA approved calaspargase pegol (Asparlas from Servier Pharmaceuticals) on 12/20/2018 to treat acute lymphoblastic leukemia as part of a multi-drug regimen. The FDA approved ravulizumab (Ultomiris from Alexion Pharmaceuticals) on 12/21/2018 for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH). The FDA approved tagraxofusp (Elzonris from Stemline Therapeutics) on 12/21/2018 for the treatment of blastic plasmacytoid dendritic cell neoplasms. The FDA delayed the PDUFA date for solriamfetol by three months to 5/20/2019 to have more time to review draft labeling submitted by Jazz. Apellis halted enrollment in the DERBY and OAKS Phase III APL-2 trials in October 2018 due to the occurrence noninfectious inflammation. An investigation by Apellis determined the cause to be an impurity or contaminant from a change in the manufacturing process after Phase II trials. A correction has been made and Apellis plans to resume both trials in 2019. The FDA designated APL-2 as an orphan drug. DBV withdrew its NDA for Viaskin Peanut to correct FDA deficiencies in its manufacturing procedures and quality control. Nabriva submitted an NDA for oral and IV lefamulin in December 2018. Serepta complete an NDA submission for golodirsen in December 2018. Aimmune submitted a BLA for AR101 in December 2018 and plans to file an MAA in 2019. Menarini licensed pracinostat from the Helsinn Group to promote and distribute the drug. Helsinn will continue to be responsible for development and manufacturing pracinostat. Trial Updates In a 6-week, 59 patient, Phase II trial, treatment with nabiximols resulted in an improvement of 0.11 points on the Modified Ashworth Scale compared to a decrease of 0.16 with placebo in patients with amyotrophic lateral sclerosis. In a 16-week, 75 patient, Phase IIa trial, hepatic fat fraction was reduced with pegbelfermin 10 mg daily (-6·8% vs -1·3%) and pegbelfermin 20 mg weekly (-5·2% vs -1·3) compared to placebo in patients with non-alcoholic steatohepatitis. Chugai announced that in the 18-month, 83 patient, Phase III SAkuraSky trial, 91.5% of patients treated with satralizumab were relapse free at 48 and 96 weeks compared to 59.9% and 53.3% with placebo in patients with neuromyelitis optica spectrum disorder that were receiving immunosuppressive therapy. Chugai announced that in the 18-month, 90 patient, Phase III SAkuraStar trial, treatment with satralizumab increased the time to relapse compared to placebo in patients with neuromyelitis optica spectrum disorder. The FDA designated satralizumab as a breakthrough therapy. Novartis has the ongoing Phase III STRIVE study evaluating intravenous AVXS-101 in spinal muscular atrophy (SMA) Type 1, 2 & 3 and Phase 1 STRONG study evaluating AVXS-101 in SMA Type 2. AVXS-101 will compete with nusinersen. Novartis bought AveXis in April 2018. The FDA accepted the BLA for AVXS-101 in December 2018. Novartis indicated it may price AVXS-101 at $4-5 million or more for the single dose treatment of SMA, but an analysis by ICER estimated a cost of $2 million would result in an estimated cost of $247,000 per QALY gained and the estimated cost per LY gained of $177,000 in patients with symptomatic Type I SMA. Clinuvel announced that in a 6-month, 18 patient, Phase trial, afamelanotide administered monthly plus ultraviolet B therapy (NB-UVB) administered twice a week resulted in follicular repigmentation in all body areas, except the feet in Asian patients with vitiligo. Patients received 6 months of afamelanotide and 7 months of NB-UVB (day 196). Pigmentation was maintained through day 280. Three patients dropped out of small Phase II trial due to concerns over darker constitutional skin after drug administration. AZ announced that 52-week, 2,133 patient Phase III ROCKIES trial, treatment with roxadustat improved hemoglobin in weeks 26-52 compared to placebo in patients with anemia from chronic kidney disease. The FDA granted Orphan Drug status to TauRx’s LMTX for the treatment of frontotemporal dementia. LMTX is a small molecule Tau aggregation inhibitor. LMTX failed to demonstrate a cognitive improvement over placebo in two phase III trials.
The FDA accepted the NDA for Intra-Cellular Therapies’ lumateperone for Acute and residual schizophrenia, in mid-December 2018 and set a PDUFA Date of 9/27/19. The drug has Fast Track priority designation. It’s a dopamine receptor phosphoprotein modulator (DPPM) with selective action on dopamine D2 and D2 receptors, along with serotonin 5-HT2A receptors. The PDUFA Date for Shield Therapeutics’ ferric maltol (Feraccru) is 7/27/19. Amgen dropped co-development rights for axalimogene filolisbac in December 2018. Axovant announced that in a 34-patient, 28-day, Phase II trial, nelotanserin did not reduce REM sleep behavior disorder compared to placebo in patients with dementia with Lewy bodies. Axovant has ceased development of nelotanserin for the treatment of dementia with Lewy bodies after the drug missed the primary endpoint. In a 128 patient, Phase II trial, AbbVie’s veliparib added to cisplatin and etoposide did not improve progression-free survival compared to placebo (6.1 vs 5.5 months) in patients with extensive-stage small-cell lung cancer. Additionally, in a 130 patient, Phase II trial, veliparib added to FOLFIRI with or without bevacizumab did not improve progression-free survival compared to placebo (12 vs 11 months) in patients with metastatic colorectal cancer. Marinus announced that in a 6-month, 11 patient, Phase II trial, treatment with ganaxolone resulted in a 54% reduction in seizure frequency in patients with PCDH19-related pediatric epilepsy who were positive for the neurosteroid allopregnanolone-sulfate (Allo-S) biomarker. In 4 patients that were negative for Allo-S, there was a 247% increase in seizure frequency. Marinus announced topline data from the 34-day, 58 patient, Phase II MAGNOLIA trial, where treatment with a 60-hour infusion of ganaxolone decreased the HAM-D17 score more than placebo by 4.2 points at 60 hours and 4.1 points at 34 days in 10 patients with postpartum depression that received the highest dose (140 µg/kg/h). Marinus is developing an oral form of ganaxolone and has an ongoing Phase II postpartum depression trial. Menlo announced that in an 8-week, 204 patient, Phase II trial, 33% of patients treated with serlopitant achieved a 4 point or > worst-itch numeric rating scale (WI-NRS) score compared to 21% with placebo in patients with pruritus associated with psoriasis. GenSight expects to report topline 48-week data from the Phase III RESCUE trial that is evaluating GS010 for the treatment of Leber’s hereditary optic neuropathy in 1Q19. XBiotech also has early trials evaluating bermekimab in the treatment of patients with moderate to severe atopic dermatitis. Mesoblast’s remestemcel-L is approved as Temcell in Japan. The company plans to submit a BLA for remestemcel-L and request accelerated review in early 2019. Novartis announced that in an 87 patient, Phase Ib dose ranging trial, treatment with alpelisib plus fulvestrant resulted in progression free survival (PFS) of 5.4 months at the maximum tolerated dose in postmenopausal women with PIK3CA-altered or PIK3CA-wild-type ER+ ABC breast cancer that progressed during or after antiestrogen therapy. PFS was longer in patients with PIK3CA-altered vs PIK3CA-wild-type tumors. The FDA approved 46 novel drugs in 2017. This made news, since it was the highest number of drugs approved in a year since 1996. The FDA surpassed the 2017 total in October 2018, with 47 drugs being approved since the beginning of the year. In November 2018, eight more drugs were approved, raising the 2018 total to 55. This surpassed the all-time high of 53 novel drug approvals established in 1996.
Over three-quarters of the drugs had priority review status. All 55 drugs met their PDUFA goal date and 39 were approved in the US before other countries. If the FDA stays with the average of 5 approvals per month, it will reach 60 by the end of the year. Leading the way in approvals this year have been 32 orphan drugs, those budget buster specialty pharmacy products that play havoc with our drug spend. 2018 is the first year that more than half of the approved new drugs had orphan indications. It is interesting to note that the number of orphan drugs approved is more than the total number of drugs approved in three of the past seven years. The Prescribe Right Pharmaceutical Pipeline Tracker is tracking three drugs with PDUFA dates at the end of December:
See how you can easily track investigational drugs with priority review status. Check out how the Pharmaceutical Pipeline Tracker can provide valuable clinical formulary decision support for Specialty Pharmacists. The FDA approved lorlatinib (Lorbrena) on 11/2/18 for the treatment of patients with ALK-positive non-small cell lung cancer (NSCLC) who have progressed on crizotinib and at least one other ALK inhibitor.
The FDA approved sufentanil sublingual tablets on 11/2/18 for the treatment of moderate-to-severe acute pain in certified medically supervised healthcare settings. The FDA approved revefenacin inhalation solution (Yupelri) on 11/9/18 for the maintenance treatment of patients with COPD. The FDA approved Salix/Cosmo Pharmaceuticals’ rifamycin (Aemcolo), on 11/17/18 for the treatment of adults with travelers’ diarrhea. The drug had FDA Qualified Infectious Disease Product and Fast Track priority designations. The FDA approved emapalumab (Gamifant from Novimmune) on 11/20/18 for the treatment of refractory, recurrent, or progressive primary hemophagocytic lymphohistiocytosis (HLH) or intolerance to conventional HLH therapy. The FDA approved glasdegib (Daurismo from Pfizer) on 11/21/18 for the treatment of newly diagnosed acute myeloid leukemia (AML) in patients aged 75 years or older or patients that are not candidates for intensive chemotherapy. The FDA approved Loxo Oncology/Bayer’s larotrectinib (Vitrakvi) on 11/26/18 for the treatment of adult and pediatric locally advanced or metastatic solid tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion regardless of site. Larotrectinib is dosed as a 100 mg capsule twice a day in adults and 100 mg/Meter-Squared orally twice daily in children. WAC for a 30-day supply of the drug will be $32,800 for adult dose capsules and start at $11,000 per month for a pediatric liquid. This is the second biomarker guided therapy after the approval of pembrolizumab last May for the treatment of microsatellite instability-high cancer in any location. Bayer and Loxo provide a value-based contract for larotrectinib that will refund payers and patients for patients who do not see a clinical benefit within the first three months of treatment. The companies will also offer patient assistance and reimbursement programs to lower patient costs. Loxo estimates there are 1,500 and 5,000 late-stage cancer patients in the U.S. and a similar number in Europe with the mutation. The FDA approved amifampridine (Firdapse) on 11/28/18 for the treatment of adults with Lambert-Eaton Myasthenic Syndrome. The FDA approved gilteritinib (Xospata) on 11/28/18 for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation. The FDA delayed a decision on Sage Therapeutics’ brexanolone by three months to work on a REMS program for the drug. The FDA did not ask for any additional safety or efficacy data. The PDUFA Date is May 19, 2019, this drug has Orphan Drug and Breakthrough Priority Designations. The FDA's Anesthetic and Analgesic Drugs Advisory Committee voted 8-7 against recommending approval of oliceridine in October 2018. The FDA rejected oliceridine in November 2018 and requested additional data on QT prolongation, a larger safety database and more nonclinical data and validation reports. The FDA’s Psychopharmacologic Drugs Advisory Committee and Drug Safety and Risk Management Committee voted 21-2 against recommending approval of Alkermes' buprenorphine/samidorphan in a joint meeting in November 2018. The committee felt that efficacy had not been proven and studies had methodological problems, but in a narrow vote (13-10) indicated that safety had been demonstrated. The FDA noted a loss of consciousness during infusion of brexanolone in 6 patients during clinical trials and recommended a REMS program and administration by a health care professional outside the patient’s home. In current data the increased risk for loss of consciousness/presyncope is 4%. The FDA’s Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee voted 17-1 this month that brexanolone demonstrated efficacy and 16-2 for safety in the treatment of postpartum depression. The FDA granted Sanofi’s Dengvaxia dengue vaccine a priority review and set a PDUFA date of 5/1/2019. The FDA has granted Fast Track designation to Karyopharm Therapeutics’ selinexor in addition to its Orphan Drug priority designation. Selinexor is intended to treat patients with diffuse large B-cell lymphoma. The drug has an April 6, 2019, PDUFA Date. The FDA has assigned Ambit Biosciences Corporation/Daiichi Sankyo’s quizartinib a PDUFA date of 5/25/19. The FDA granted Orphan Drug, Breakthrough Therapy, and Fast Track priority designations to the drug. The FDA informed CTI that a Phase III myelofibrosis trial will need to be performed before submitting an NDA for pacritinib. CTU plans to start the trial in early 2019. CTI submitted an MAA to the Committee for Medicinal Products for Human Use (CHMP). The FDA's Endocrinologic and Metabolic Drugs Advisory Committee will review sotagliflozin in mid-January. The FDA's Reproductive and Urologic Drugs Advisory Committee review romosozumab in mid-January. The FDA set a December 21, 2018, PDUFA Date for Shire/Janssen’s prucalopride. There are no priority designations for the drug.
Acceleron and Celgene announced that in the 48-week, 336 patient, Phase III BELIEVE trial, 21.4% of patients treated with luspatercept had at least 33% reduction in red blood cell (RBC) transfusions compared to 4.5% with placebo in patients with beta thalassemia. Celgene and Juno announced that in a 16 patient, Phase I trial, treatment with lisocabtagene maraleucel led to an overall response of 81% and a complete response in 43% in patients with refractory or resistant chronic lymphocytic leukemia. Apellis announced interim data from 10 patients in the Phase 1, PADDOCK trial, where APL-2 patients achieved transfusion independence with an average hemoglobin increase of 4.2 g/dL by day 85 in patients with paroxysmal nocturnal hemoglobinuria. Apellis announced interim data from autoimmune hemolytic anemia patients in the open label, Phase II, PLAUDIT trial where APL-2 increased hemoglobin by 3.4 g/dL at day 56 in 12 patients with cold agglutinin disease and by 2.0 g/dL in warm antibody autoimmune hemolytic anemia. NewLink announced that in a Phase II trial, of 102 patients with advanced melanoma, indoximod monotherapy or in combination with a checkpoint inhibitor (pembrolizumab, nivolumab or ipilumumab), 70 of the patients had an ORR of 56%. Novartis has the ongoing Phase III STRIVE study evaluating intravenous onasemnogene abeparvovec (Zolgensma) in spinal muscular atrophy Type 1, 2 & 3 and Phase 1 STRONG study evaluating onasemnogene abeparvovec in spinal muscular atrophy Type 2. Onasemnogene abeparvovec will compete with nusinersen. Novartis bought AveXis in April 2018. The FDA accepted the BLA for onasemnogene abeparvovec in December 2018 and set a PDUFA Date of 5/3/19. AveXis indicated it may price onasemnogene abeparvovec at $4 million or more for the single dose treatment of spinal muscular atrophy. Biohaven announced that in a 1,351 patient, Phase III trial, 21.2% of patients with acute migraine treated with rimegepantin an orally dissolving tablet (ODT) formulation were pain-free and 35.1% were free of their most bothersome symptoms at two hours compared to 10.9% and 26.8% that received placebo. Onconova announced that in a 74 patient, Phase II trial, treatment with the combination of rigosertib and azacitidine demonstrated an overall response rate of 90% and complete remission in 34% of patients with myelodysplastic syndrome. TG Therapeutics announced that in a 10 patient, Phase I/II trial, a combination of umbralisib, ublituximab and pembrolizumab resulted in a complete response in one patient and a partial response in 8 patients in patients with relapsed/refractory chronic lymphocytic leukemia. TG Therapeutics announced interim results from a Phase I/Ib trial where a combination of umbralisib, ublituximab and bendamustine resulted in an overall response in 12/25 patients with diffuse large B-cell lymphoma and 11/13 patients with follicular lymphoma. Redhill announced that in the 455 patient, Phase III, ERADICATE Hp2 trial, the combination of amoxicillin, omeprazole and rifabutin (RH-105) demonstrated an 84% eradication of H. pylori compared to 58% eradication with amoxicillin and omeprazole in dyspepsia patients with confirmed H. pylori infection. Redhill plans to file an NDA in the first half of 2019. Legend and J&J announced interim results from 57 patients in a phase I Chinese trial where JNJ-68284528 demonstrated an overall response rate of 88% and complete response of 74% in patients with relapsed multiple myeloma. 90% of patients in the interim analysis developed cytokine release syndrome (CRS) with 4 developing severe CRS. All but one CRS patient recovered. Pyrexia was the most common ADR. Only one (out of 57) has developed mild neurotoxicity. Bluebird announced that in a 21 patient, Phase I trial, treatment with bb2121 resulted in complete remission in 56% of patients with relapsed or refractory multiple myeloma and 9-months progression-free survival of 71%. Bluebird announced that in a 43 patient, Phase I trial, relapsed multiple myeloma patients were treated with a lymphodepleting regimen of fludarabine and cyclophosphamide followed by bb2121. Half of the patients that received a higher dose (>150,000,000 cells) of bb2121 had a PFS of 10.8 months, a CR of 50% and an ORR ion 95.5%. Bluebird announced interim results from 12 patients in a Phase I trial where treatment with bb21217 (a longer lasting version of bb2121) resulted in an 83% objective response rate in heavily pretreated multiple myeloma patients. Karyopharm announced interim results from the first 115 or 127 patients in the Phase IIb, SADAL trial, where treatment with selinexor resulted in a 29.6% Overall Response Rate in patients with relapsed or refractory diffuse large B-cell lymphoma. Karyopharm announced interim results from two arms of the Phase Ib/II STOMP trial. In one arm, selinexor in combination with daratumumab achieved an overall response rate of 73% in patients with relapsed or refractory multiple myeloma. In the second arm, selinexor in combination with pomalidomide achieved an overall response rate of 50% in patients with relapsed or refractory multiple myeloma. Karyopharm announced that in the 122 patient, Phase IIb, STORM trial, treatment with selinexor in combination with dexamethasone resulted in a 26% overall response rate in patients with highly resistant multiple myeloma. Geron announced interim results from 59 patients in the 24-week, Phase II, IMbark trial, where 10% of patients treated with imetelstat achieved a ≥35% reduction in spleen volume and 32% achieved a ≥50% reduction in Total Symptom Score, at 24 weeks in Intermediate-2 or High-risk myelofibrosis patients who are relapsed or refractory to a Janus Kinase inhibitor. Global Blood Therapeutics announced that in the 24-week, 154 patient Phase III, HOPE trial, 65% of voxelotor 1,500 mg patients and 33% of the 900 mg patients had a 1 g/dL increase in hemoglobin compared to 10% with placebo in patients with sickle cell disease. AbbVie announced it was stopping enrollment in the Phase III TAHOE small cell lung cancer trial after an independent interim review found that treatment with rovalpituzumab resulted in worse overall survival than the comparator topotecan. AbbVie will continue development of rovalpituzumab for other indications but will not seek accelerated approval. Cerenis announced that in the 24-week, Phase III, TANGO trial, CER-001 did not reduce mean vascular wall area compared to placebo in patients with Familial Hypoalphalipoproteinemia. Conatus announced that in a 24-week, 263 patient, Phase IIb trial, emricasan did not improve hepatic venous pressure gradient compared to placebo in patients with nonalcoholic steatohepatitis. Genfit announced that in a 12-week, 45 patient, Phase II trial, treatment with elafibranor resulted in a 48% decrease in serum alkaline phosphatase with 80 mg and a 41% decrease with 120 mg compared to a 3% increase with placebo in patients with primary biliary cholangitis that had an inadequate response to ursodeoxycholic acid. Immunomedics announced that in a 108 patient, Phase II clinical trial, treatment with sacituzumab govitecan resulted in an ORR of 33% and a duration of response of 7.7 months in patients with triple-negative breast cancer. Spectrum announced that in 237 patient, Phase III RECOVER trial, eflapegrastim was non-inferior to pegfilgrastim in reducing the duration of severe neutropenia during four cycles of treatment in patients with breast cancer. Tralokinumab failed to decrease annual exacerbation in severe asthmatics compared to placebo in the 52-week, 1,000 patient, Phase III STRATOS-1 trial and a 454-patient, Phase IIb trial. AstraZeneca announced topline data from the Phase III, STRATOS 2 trial of asthma patients with increased IL-13 activity, where treatment with tralokinumab did not reduce exacerbations compared to placebo. In the 12-week, 224 patient, Phase II, MESOS trial, treatment with tralokinumab did not lower bronchial eosinophil count compared to patients with moderate to severe asthma that was inadequately controlled on inhaled corticosteroids. In the 40-week, 140 patient, Phase III, TROPOS trial of asthma patients with increased IL-13 activity, treatment with tralokinumab did not reduce the need for corticosteroids compared to placebo. AstraZeneca announced that in the Phase III, open-label, EAGLE trial, neither durvalumab monotherapy or durvalumab plus tremelimumab improved overall survival compared to standard-of-care chemotherapy in patients with platinum-resistant head and neck squamous cell carcinoma. On Tuesday’s update we noted that Joanne Stubbings, B.S. Pharm., MHCA, announced the creation of the Section of Specialty Pharmacy Practitioners. It’s the first new section created in twelve years. Three thousand AHSP members have already registered, representing a cross section of health system pharmacy. The section’s mission is to advance the cause of health system-based specialty pharmacy practice. Three advisory groups have been created: workforce development, business development, and outcomes and values.
The Board of Pharmacy Specialties notes that “With specialty-trained pharmacists as part of the collaborative care team, enhanced patient satisfaction has been documented with fewer complications in drug treatment; improved laboratory monitoring; reductions in unnecessary medications; and shorter hospital stays resulting in lower treatment costs. BPS currently provides board certification in Ambulatory Care, Nuclear Pharmacy, Nutrition Support, Oncology, Pharmacotherapy and Psychiatric Pharmacy. Certification exams for Critical Care and Pediatric Pharmacy are in development.” APhA defines Specialty pharmacy as focusing on the provision of high cost, high touch medication therapy for patients with complex disease states. Medications in specialty pharmacy range from oral to cutting edge injectable and biologic products. The disease states treated range from cancer, multiple sclerosis and rheumatoid arthritis to rare genetic conditions. The Pharmaceutical Pipeline Tracker can provide valuable clinical formulary decision support for Specialty Pharmacists. Investigational drugs in the above-mentioned disease states are covered comprehensively in our knowledgebase. The Pharmaceutical Pipeline Tracker helps to manage the disruptive impact of the launch of new specialty drugs on formulary management and provides actionable clinical decision support. For example, there are twenty-one different therapeutic categories and one hundred forty-seven investigational cancer drugs in our knowledgebase. The FDA approved Loxo Oncology/Bayer’s larotrectinib (Vitrakvi) on 11/26/18 for the treatment of adult and pediatric locally advanced or metastatic solid tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion regardless of site. Larotrectinib is dosed as a 100 mg capsule twice a day in adults and 100 mg/Meter-Squared orally twice daily in children. WAC for a 30-day supply of the drug will be $32,800 for adult dose capsules and start at $11,000 per month for a pediatric liquid. This is the second biomarker guided therapy after the approval of pembrolizumab last May for the treatment of microsatellite instability-high cancer in any location. Bayer and Loxo provide a value-based contract for larotrectinib that will refund payers and patients for patients who do not see a clinical benefit within the first three months of treatment. The companies will also offer patient assistance and reimbursement programs to lower patient costs. Loxo estimates there are 1,500 and 5,000 late-stage cancer patients in the U.S. and a similar number in Europe with the mutation. There are twelve investigational drugs targeting Multiple Sclerosis in our knowledgebase. For example, Novartis’ siponimod has a PDUFA Date of March 31, 2019. The drug has no FDA Priority Designations. There are links to six studies in the knowledgebase to fully functional MedLine monographs. At this point in time, none of the other eleven MS drugs under development have PDUFA dates. We’ve aggregated a large knowledgebase of investigational specialty drugs. One may choose to access information about these drugs by using one of three methodologies: by Therapeutic Category, by PDUFA Date, or by Indication. Click on each search type to link to more information about each methodology. Exciting news from ASHP Mid-Year. Joanne Stubbings, B.S. Pharm., MHCA, announced the creation of the Section of Specialty Pharmacy Practitioners. It’s the first new section created in twelve years. Three thousand AHSP members have already registered representing a cross section of health system pharmacy. The section’s mission is to advance the cause of health system-based specialty pharmacy practice. Three advisory groups have been created: workforce development, business development, and outcomes and values.
The FDA approved Loxo Oncology/Bayer’s larotrectinib (Vitrakvi) on 11/26/18 for the treatment of adult and pediatric locally advanced or metastatic solid tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion regardless of site. Larotrectinib is dosed as a 100 mg capsule twice a day in adults and 100 mg/Meter-Squared orally twice daily in children. WAC for a 30-day supply of the drug will be $32,800 for adult dose capsules and start at $11,000 per month for a pediatric liquid. This is the second biomarker guided therapy after the approval of pembrolizumab last May for the treatment of microsatellite instability-high cancer in any location. Bayer and Loxo provide a value-based contract for larotrectinib that will refund payers and patients for patients who do not see a clinical benefit within the first three months of treatment. The companies will also offer patient assistance and reimbursement programs to lower patient costs. Loxo estimates there are 1,500 and 5,000 late-stage cancer patients in the U.S. and a similar number in Europe with the mutation. The FDA approved amifampridine (Firdapse) on 11/28/18 for the treatment of adults with Lambert-Eaton Myasthenic Syndrome. The FDA approved gilteritinib (Xospata) on 11/28/18 for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation. The FDA has assigned Ambit Biosciences Corporation/Daiichi Sankyo’s quizartinib a PDUFA date of 5/25/19. The FDA granted Orphan Drug, Breakthrough Therapy, and Fast Track priority designations to the drug. The FDA informed CTI that a Phase III myelofibrosis trial will need to be performed before submitting an NDA for pacritinib. CTU plans to start the trial in early 2019. CTI submitted an MAA to the Committee for Medicinal Products for Human Use (CHMP). The FDA's Endocrinologic and Metabolic Drugs Advisory Committee will review sotagliflozin in mid-January. The FDA's Reproductive and Urologic Drugs Advisory Committee review romosozumab in mid-January. In a Phase III trial, patients with obstructive sleep apnea treated with Jazz’s solriamfetol maintained their improvement through 4 weeks in the Maintenance of Wakefulness Test and Epworth Sleepiness Scale at 6 weeks compared to worsening with placebo in patients with excessive sleepiness. It was a busy news week for Alkermes.
J&J announced that in a Phase III, rheumatoid arthritis trial, ACR20 was achieved at 1-year by 76.9% of patients treated with sirukumab 50 mg every 4 weeks and 76.9% treated with 100mg every 2 weeks. After two years, 68.3% of patients treated with sirukumab 50 mg every 4 weeks and 74.6% treated with 100mg every 2 weeks maintained ACR20. In a non-randomized, non-controlled, Phase III safety study, Urovant/Kyorin/Merck’s vibegron improved daily mean micturitions, urgency episodes, urgency incontinence episodes, incontinence episodes and night-time frequency from baseline in patients with overactive bladder. In another Phase III trial, vibegron improved daily mean micturitions, urgency episodes, urgency incontinence episodes, incontinence episodes and night-time frequency compared to placebo in patients with overactive bladder. In a Phase II trial, 46% of patients treated with Acacia Pharma’s amisulpride had no emesis or use of rescue medication compared to 59% with a standard triple therapy regimen and 20% with placebo in patients at risk for the delayed phase of chemotherapy-induced nausea and vomiting in chemotherapy-naive patients receiving cisplatin or an anthracycline-cyclophosphamide regimen for breast cancer. Karyopharm announced interim results from the first 115 or 127 patients in the Phase IIb, SADAL trial, where treatment with selinexor resulted in a 29.6% Overall Response Rate in patients with relapsed or refractory diffuse large B-cell lymphoma. Stay current with drugs in the late stages of development with the Prescribe Right Pharmaceutical Pipeline Tracker. |
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