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Pipeline News and Updates
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Weekly Update for the Week Ending December 21, 2019

12/30/2019

 
Regulatory Update

The FDA approved for lemborexant (Dayvigo, Eisai) on 12/23/19 for the treatment of insomnia characterized by difficulties with sleep onset and/or sleep maintenance.

The FDA approved for lumateperone (Caplyta, Intra-Cellular Therapies) on 12/23/19 for the treatment of schizophrenia in adults. Lumateperone has a boxed warning due to an increased risk of death for elderly patients with dementia-related psychosis. The label also contains a warning for increased risk for cerebrovascular events in this population.

The FDA approved ubrogepant (Ubrelvy, Allergan) on 12/23/19 for the acute treatment of migraine with or without aura in adults.

Announced Research Updates

Shanghai Green Valley Pharmaceutical plans to initiate an 18-month, 2,046 patient, Phase III trial in the U.S. and Europe in 2020 that will evaluate oligomannate in the treatment of mild-to-moderate Alzheimer's disease. Results were announced in the fall of 2019 from a 9-month trial, which found oligomannate slowed the decrease in the ADAS-Cog12 Score compared to placebo. While researchers were skeptical due to the short duration of the trial and lack of details, the results led to conditional approval in China. The new trial is being undertaken to answer questions about the drug from the FDA and EMA.

Published Research Updates

In a 312 patient, Phase III trial,  pixantrone/rituximab did not improve progression free survival compared to gemcitabine/rituximab in patients with diffuse large B-cell lymphoma (DLBCL) or follicular lymphoma (FL) grade 3 who relapsed after ≥1 rituximab-containing regimen and were not eligible for a stem cell transplant.

The World Health Organization recommended fexinidazole as a first-line treatment option for human African trypanosomiasis. The Committee for Human Medicinal Products (CHMP) of the European Medicines Agency (EMA) recommended approval of fexinidazole for the treatment of human African trypanosomiasis (HAT), more commonly known as sleeping sickness. CHMP gave the opinion under Article 58, which is designed to provide guidance for drugs that are intended for use in countries outside the European Union. Fexinidazole was approved for use in the Democratic Republic of the Congo.

Weekly Update for the Week Ending December 21, 2019

12/23/2019

 
Regulatory Update
 
The FDA approved trastuzumab deruxtecan (Enhertu, AstraZeneca/Daiichi Sankyo) on 12/19/19, for the treatment of unresectable or metastatic HER2-positive breast cancer that has received two or more prior anti-HER2-based regimens in the metastatic setting.
 
The FDA approved Merck’s Ebola virus vaccine (Ervebo) on 12/20/19, for the prevention of Ebola virus disease caused by the Zaire ebola virus in people at least 18 years old.
 
The FDA approved enfortumab vedotin (Padcev, Astellas/Seattle Genetics) on 12/18/19 for the treatment of locally advanced or metastatic urothelial cancer that has progressed on chemotherapy and immunotherapy.
 
The FDA rejected ViiV Healthcare/Janssen’s NDA for the combination of cabotegravir and rilpivirine for HIV treatment, due to manufacturing issues.
 
The FDA’s review of tazemetostat found a lack of efficacy compared to standard therapies and the potential for secondary malignancies that short trial durations may not have fully described. But the FDA’s Oncologic Drugs Advisory Committee voted 11-0 with two abstentions to recommend approval of tazemetostat for the treatment of metastatic or inoperable locally advanced epithelioid sarcoma, due to the low response rate with currently approved treatments.
 
Deciphera filed an NDA for ripretinib for the treatment of gastrointestinal stromal tumors.
 
The FDA designated Orphazyme’s arimoclomol a Fast Track drug for the treatment of sporadic Inclusion Body Myositis.
 
PharmaMar submitted an NDA for lurbinectedin for the treatment of small cell lung cancer. 
 
The FDA designated Cascadian Therapeutics’ tucatinib a Breakthrough Therapy when given in combination with trastuzumab and capecitabine, for the treatment of patients with locally advanced unresectable or metastatic HER2-positive breast cancer.
 
BMS filed a BLA for lisocabtagene maraleucel for the treatment of relapsed or refractory large B-cell lymphoma (LBCL) after at least two prior therapies.
 
MacroGenics filed a BLA for margetuximab for the treatment of patients with metastatic HER2-positive breast cancer in combination with chemotherapy.
 
Gilead submitted an NDA for filgotinib for the treatment of moderate-to-severe rheumatoid arthritis.
 
Announced Research Updates
  1. Gilead announced that in the 48-week, 392 patient, Phase II ATLAS study, liver fibrosis was not improved by 1 stage or more without disease worsening compared to placebo in patients treated with selonsertib, cilofexor or firsocostat alone or in two-drug combinations in patients with advanced fibrosis (F3-F4) due to NASH.
  2. BioMarin announced that in a 52-week, 121 patient, Phase III trial, treatment with vosoritide resulted in an increase of 1.6 cm/year in annualized growth velocity compared to placebo in children (ages 5 to 14) with achondroplasia.
  3. Novartis announced that in two 52-week, Phase III trials (LUSTER 1 and LUSTER 2) that enrolled a total of 1,771 patients, treatment with fevipiprant did not reduce moderate -to-severe exacerbation compared to placebo in patients with inadequately controlled moderate-to-severe asthma. Novartis has stopped development of fevipiprant for the treatment of asthma.
  4. Proxel announced that in the 52-week, 714 patient, Phase III, open-label, TIMES 2 trial, imeglimin reduced HbA1c 0.12 to 0.92 when added to non-insulin anti-diabetic drugs in Japanese patients with type 2 diabetes.
  5. Lexicon announced that in the 26-week, 700 patient, Phase III SOTA-EMPA trial, treatment with sotagliflozin was superior to placebo and non-inferior to empagliflozin in reducing HbA1c in patients with type 2 diabetes and inadequate glycemic control on a DPP4 inhibitor with or without metformin.
Published Research Updates
  1. In a 1,140 patient, Phase III trial, veliparib added to carboplatin and paclitaxel followed by veliparibmaintenance therapy improved progression-free survival (PFS) compared to carboplatin plus paclitaxel induction therapy alone (23.5 months vs 17.3 months) in patients with previously untreated stage III or IV high-grade serous ovarian carcinoma. In patients with a BRCA-mutation the difference in PFS was 34.7 months with veliparib compared to 22 months with placebo.
  2. In a 73-patient, Phase I, open-label trial, treatment with belantamab mafodotin resulted in a 33% overall response rate in patients with multiple myeloma. In the 196-patient, Phase II, open-label, DREAMM-2 trial, treatment with belantamab mafodotin resulted in a 48% overall response rate in patients with multiple myeloma.
  3. A 52-week, 701 patient, Phase III trial was terminated early, when treatment with mongersen did not improve the clinical remission rate compared to placebo at 12-weeks in patients with Crohn's disease.

Weekly Update for the Week Ending December 14, 2019

12/16/2019

 
Regulatory Update
 
The FDA approved golodirsen (Vyondys 53, Sarepta Therapeutics) on 12/12/2019 for the treatment of Duchenne muscular dystrophy amenable to exon 53 skipping. We took a look at this surprise reversal of the August 2019 rejection of the drug in a post last week.
 
The FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee voted 12-0 to recommend approval of Horizon Therapeutics’ teprotumumab in December 2019 for the treatment of Thyroid Eye Disease.
 
The FDA lifted the clinical trial hold for Abeona Therapeutics’ EB-101 targeting recessive dystrophic epidermolysis bullosa
 
The FDA designated Dermira/Roche’s lebrikizumab a Fast Track treatment for moderate-to-severe atopic dermatitis.
 
The FDA Cardiovascular and Renal Drugs Advisory Committee voted 11-2 against recommending approval of Correvio’s vernakalant due to safety concerns over hypotension, arrhythmias, and conduction disturbance.
 
Biocryst submitted an NDA for BCX7353 in December 2019 with a hereditary angioedema indication.
 
Seattle Genetics plans to file an NDA and MAA for tucatinib in 1Q20.
 
Announced Research Updates
  1. Bluebird Bio announced that in the 5-year, 23 patient, Phase I/II, open-label Northstar Trial, treatment with elivaldogene tavalentivec resulted in transfusion independence (Hemoglobin of at least 9 g/dL and no transfusions for more than 12 months) in 8 out of 10 beta-thalassemia patients without the beta 0/beta 0 genotype and 3 out of 8 who did. 
  2. Iterum Therapeutics announced that in the 668 patient, Phase III SURE 3 trial, IV sulopenem followed by oral sulopenem/probenecid did not meet non-inferiority compared to to IV ertapenem followed by oral ciprofloxacin/metronidazole or amoxicillin-clavulanate in the treatment of complicated intra-abdominal infections.
  3. Leo Pharma announced the results from three Phase III trials (ECZTRA 1, 2 and 3) involving 1,976 patients with moderate-to-severe atopic dermatitis who were candidates for systemic therapy. In the trials more patients treated with tralokinumab with or without topical corticosteroids achieved an Investigator Global Assessment (IGA) score of clear (0) or almost clear (1) skin at week 16 and at least a 75% or > improvement in their Eczema Area and Severity Index (EASI) score at week 16 compared to placebo. The FDA has given tralokinumab and orphan drug priority designation.
  4. MacroGenics announced interim data from the 563 patient, Phase III, SOPHIA trial, where margetuximab plus one of four chemotherapy agents (capecitabine, eribulin, gemcitabine or vinorelbine) resulted in overall survival of 21.6 months compared to 19.8 months with trastuzumab and one of four chemotherapy agents in previously treated HER-2 positive metastatic breast cancer patients who were receiving standard chemotherapy. There was no difference in progression-free survival between the two drugs.
  5. Mustang Bio announced updated interim data from 11 XSCID infants that completed 23.6-months in a Phase I/II, open-label trial, suggested that treatment with MB-107 lead to normal for age levels of NK cells in 3-4 months in 9 patients.
  6. Cara announced that in a 12-week, Phase II trial, treatment with oral difelikefalin 1 mg improved the daily 24-hour Worst Itching Intensity Numeric Rating Scale (WI-NRS) score compared to placebo (-4.4 vs -3.3) in patients with CKD-associated pruritus.
Published Research Updates
  1. In the 24-hour, 768 patient, Phase III, open-label ATHENA trial, treatment with Travena’s oliceridine reduced the NRS pain score by 2.2 points at 30 mins from a baseline score of 6.3 in surgical (94%) and non-surgical patients with moderate to severe acute pain.
  2. In the 184 patient, Phase II, open-label, DESTINY-Breast01 trial, treatment with AstraZeneca/ Daiichi Sankyo’strastuzumab deruxtecan resulted in an objective response rate of 60.9% of patients with HER2 positive metastatic breast cancer previously treated with trastuzumab emtansine.
  3. In a 21-week, 38-patient, Phase II trial, 46% of patients treated with Argenx’ efgartigimod added to standard of care therapy improved their platelet count to 50x109/L during two or more visits compared to 25% in the placebo cohort in patients with primary immune thrombocytopenia (ITP).
  4. A 52-week, 172 patient, open-label extension of a 14-week efficacy trial examined the safety for Daiichi Sankyo’s mirogabalin and found the most common adverse effect to be nasopharyngitis, diabetic retinopathy, peripheral edema, somnolence, diarrhea, increased weight, and dizziness in Asian patients with diabetic peripheral neuropathic pain.
  5. ​In the 1-year, 480 patient, Phase II, HER2CLIMB trial, treatment Seattle Genetics tucatinib in combination with trastuzumab and capecitabine resulted in progression-free survival in 33.1% of patients compared to 12.3% with trastuzumab and capecitabine alone in patients with locally advanced unresectable or metastatic HER2-positive breast cancer who were previously treated with trastuzumab, pertuzumab and trastuzumab emtansine.

Surprise Turnaround: Golodirsen is Approved

12/13/2019

 
The FDA approved golodirsen (Vyondys 53, Sarepta Therapeutics) on 12/12/2019 for the treatment of Duchenne muscular dystrophy amenable to exon 53 skipping. This is a reversal of the August 2019 decision, where the FDA rejected golodirsen due to the risk of infection with intravenous infusion ports and renal toxicity seen in pre-clinical studies. 

Sarepta plans to price  golodirsen similar to eteplirsen. The average annual patient cost for eteplirsen is $300,000, but the cost can exceed $1 million because the drug is dosed by weight. A review by ICER of treatments for Duchenne’s found insufficient data that a slight increase in levels of dystrophin with eteplirsen and golodirsen provides a clinically significant effect. Due to the lack of evidence supporting a clinical benefit with the drugs, ICER did not estimate the pharmacoeconomic impact of either drug. 

Sarepta has shown that golodirsen will increase dystrophin levels, but has not completed studies to demonstrate an improvement in muscle function. The FDA is requiring Sarepta to complete a clinical efficacy study, so the company is recruiting at least 45 boys for the Phase III ESSENCE trial that will compare golodirsen to placebo on the impact of a 6-minute walking test. The trial is projected to be completed in 2024.

Weekly Update for the Week Ending December 7, 2019

12/10/2019

 
Regulatory Update
 
The FDA rejected Lexicon Pharmaceuticals/Sanofi’s sotagliflozin a second time as a treatment for type 1 diabetes on 12/2/2019. Previously, the FDA's Endocrinologic and Metabolic Drugs Advisory Committee had a split decision (8-8) on sotagliflozin on whether to recommend approval of the drug as an add-on to insulin therapy in patients with type 1 diabetes in January 2019. Some members of the committee expressed concern regarding the increased risk for diabetic ketoacidosis with the drug. The FDA rejected sotagliflozin as a treatment for type 1 diabetes on 3/22/2019. The EMA approved sotagliflozin in April 2019 for the treatment of Type 1 Diabetes. Sanofi ceased participation in the development of sotagliflozin in July 2019 after the results from 3 Phase III type 2 diabetes trials were announced. 
 
The FDA rejected RVT-802 on 12/5/19 due to manufacturing issues.
 
Immunomedics resubmitted the BLA for sacituzumab govitecan for the treatment of resistant metastatic triple-negative breast cancer.
 
ViiV Healthcare submitted an NDA for fostemsavir in December 2019. Fostemsavir was bought from BMS in December 2015.
 
The FDA has placed a partial clinical hold on four of Clementia’s palovarotene trials [(two fibrodysplasia ossificans progressiva (FOP) studies and two osteochondroma studies)] due to reports of early growth plate closure in patients with FOP in patients under the age of 14.
 
Announced Research Updates
  1. AB Science announced that in a 420 patient, Phase III trial, treatment with masitinib resulted in a decrease in severe asthma exacerbations compared to placebo in patients with severe asthma uncontrolled by oral corticosteroids.
  2. Correvio announced that an analysis of the European SPECTRUM registry evaluated 1,289 episodes of recent-onset symptomatic AF in 1,120 ED patients with atrial fibrillation, where a single infusion of vernakalant converted 61% of cases to sinus rhythm and 70.2% of cases were converted that received a second dose. The analysis will be reviewed by the FDA's Cardiovascular and Renal Drugs Advisory Committee today, 12/10/2019.  Vernakalant has a PDUFA date this month of 12/24/2019. Vernakalant was rejected by the FDA in 2008, but approved in Europe and Canada in 2010. The SPECTRUM registry was initiated in 2010 at the request of the European Medicines Agency as a follow-up measure.
  3. Aldeyra announced that in part 1 of the 12-week, 422 patient, Phase III, RENEW trial, dosing reproxalap four-times-daily for four weeks, followed by twice-daily dosing improved patient-reported ocular dryness score compared to placebo, but continuous dosing of the drugs four-times-daily did not improve symptoms compared to placebo in patients dry eye disease. Neither dosing regimen improved fluorescein nasal region staining score compared to placebo.
  4. Sage Therapeutics announced that in the 15-day, 581 patient, Phase III, MOUNTAIN trial, treatment with zuranolone did not improve the HAMD-17 score compared to placebo (12.6 vs 11.2) in patients with major depressive disorder.
  5. Aurinia announced that in a 52-week, 357 patient, Phase III, AURORA trial, 40.8% of patients treated with voclosporin achieved complete renal remission (UCPR of 0.5 mg/mg or <, eGFR 60 mL/min/1.73 m2 or < with no decrease of > 20% and no continuation of low dose corticosteroids or use of rescue medication) compared to 22.5% with placebo in lupus nephritis patients treated with mycophenolate mofetil and rapidly tapered low-dose oral corticosteroids. Aurinia plans to submit an NDA for voclosporin in the first half of 2020.
  6. MyoKardia announced 48-week interim data from 12 patient enrolled in the Phase II extension of the PIONEER-HCM trial (PIONEER-OLE), suggested that 9/12 were asymptomatic and improvements in biomarkers, left ventricular ejection fraction and left ventricular outflow tract gradient were maintained at 48 weeks.
  7. FerGene announced that in a 157 patient, Phase III, open-label trial, 53% of patients treated with nadofaragene firadenovec had a complete response at 3 months and 24% at 12 months in patients with BCG unresponsive non-muscle invasive bladder cancer.
  8. UCB announced that in the 16-week, 478 patient, Phase III, BE SURE trial, more patients treated with bimekizumab achieved PASI 90 and an IGA score of clear or almost clear than adalimumab in patients with moderate-to-severe chronic plaque psoriasis. UCB plans submit for bimekizumab to treat moderate-to-severe plaque psoriasis in mid-2020.
  9. Epizyme announced interim data from 99 patents enrolled in a Phase II study that suggested treatment with tazemetostat resulted in an overall response rate of 69% (31/45) in patients with EZH2 activating mutations follicular lymphoma and 35% (19/54) in patients with wild-type EZH2 follicular lymphoma.
  10. Biohaven Pharmaceutical announced that interim data from 100 patients, who had completed 6-months of treatment in the Phase II/III, T2 Protect AD Study trial, suggested that treatment with troriluzole improved cognitive function or hippocampal volume in patients with mild-to-moderate Alzheimer's disease.
  11. BMS announced that in the 269 patient, Phase I, TRANSCEND trial, treatment with lisocabtagene maraleucel resulted in a 73% overall response rate and a 53% complete response rate in patients with relapsed or refractory large B-cell lymphoma (LBCL). And in the 22 patient, Phase I/II, TRANSCEND CLL trial, treatment with lisocabtagene maraleucel resulted in a 81.5% overall response rate and a 45.5% complete response rate in patients with high-risk chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
  12. ObsEva announced that in the 535 patient, Phase III PRIMROSE 2 trial, heavy menstrual bleeding was reduced to 80/ml or less with the reduction being at least 50% from baseline in 93.9% of patients treated with linzagolix 200 mg and hormone add-back therapy and 56.7% with linzagolix 100 mg monotherapy compared to 29.4% with placebo in patients with uterine fibroids and heavy menstrual bleeding. The Phase III PRIMROSE 1 uterine fibroid trial is due to be completed in 2Q20. If the results for PRIMROSE 1 are positive, ObsEva plans to submit an MAA for linzagolix for the treatment of heavy menstrual bleeding associated with uterine fibroids by the end of 2020 and an NDA in early 2021.
  13. J&J announced that in a 29 patient, Phase Ib CARTITUDE-1 trial, JNJ-4528 demonstrated an overall response rate of 100% and complete response of 69% in patients with relapsed multiple myeloma. 
Published Research Updates
  1. A pooled analysis of six Phase II trials involving 103 patients found that midomafetamine (MDMA) added to psychotherapy resulted in 54.2% of patients not meeting CAPS-IV PTSD diagnostic criteria compared to 22.6% in the placebo group in patients with PTSD.
  2. In a 12-week, 1,001 patient, Phase III trial, treatment with esaxerenone was noninferior to eplerenone for reductions in sitting and 24-hour BP in Japanese patients with essential hypertension. 
  3. In the 1,672 patient, ACHIEVE I trial, the percentage of patients, who were pain free at two hours was 19.2% with ubrogepant 50 mg, 21.2% with 100 mg and 11.8% with placebo. The most bothersome symptoms at two hours were eliminated in 38.6% treated with ubrogepant 50 mg, 37.7% with 100 mg and 27.8% with placebo.
  4. In a 32 patient cohort from a Phase II, advanced solid tumor, open-label trial, 18.8% of patients treated with tremelimumab had an objective response in patients with metastatic urothelial cancer.​
  5. In the 350 patient, Phase III, TIVO-3 trial, patients treated with tivozanib had a median progression-free survival of 5.6 months compared to 3.9 months with sorafenib in patients with with highly refractory advanced or metastatic renal cell carcinoma that had failed at least two prior therapies, including a vascular EGFR TKI.

FDA Approval Surge

12/5/2019

 
During October and November 2019, we saw the FDA approve thirteen new investigational drugs. Five of these drugs have annual costs in excess of $100,000. Each of these potential budget busters was approved months ahead of their PDUFA Dates, targeting such indications as the most common cystic fibrosis mutation, mantle cell lymphoma for patients who have received at least one prior therapy, sickle cell disease, partial-onset seizures in adults, and complicated urinary tract infections.

Here’s a link to a relevant article about the five budget busters from the November 27th ENDPOINTS News about approval process, Congress’ viewpoint and what FDA is analyzing https://endpts.com/fda-approves-5-new-costly-drugs-well-ahead-of-pdufa-dates/

Thirteen October and November Approvals

  1. The FDA approved trifarotene cream (Aklief, Galderma) on October 4 for the treatment of acne vulgaris.
  2. The FDA approved lasmiditan (Reyvow, Lilly) on 10/11/2019 for the treatment of acute migraines, with or without aura, in adult patients. Due to the occurrence of dizziness and sedation from CNS depression, patients are warned of potential impairment while taking lasmiditan and advised not to drive, operate machinery or take with alcohol or other CNS depressants for at least eight hours after taking the drug, even if they feel well enough to do so. The FDA announced that during clinical trials the most common ADR with lasmiditan were dizziness, fatigue, paresthesia and sedation.
  3. The FDA approved brolucizumab (Beovu, Novartis) on 10/8/2019 for the treatment of wet age-related macular degeneration (AMD). Brolucizumab is administered by intravitreal injection every three months. WAC was set at $1,850 per dose, the same as aflibercept (Eylea), which is dosed more frequently. After the loading dose, analysts estimate the annual brolucizumab WAC at $16,000 for both eyes compared to $24,000 for aflibercept and and $40,000 for ranibizumab (Lucentis).
  4. The FDA approved afamelanotide (Scenesse, Clinuvel) on 10/8/2019 to treat phototoxic reactions in patients with erythropoietic protoporphyria.
  5. The FDA approved the combination of elexacaftor, ivacaftor, and tezacaftor (Trikafta, Vertex Pharmaceuticals) on October 21 for the treatment of patients age 12 years or older with cystic fibrosis (CF) and at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. The combination was approved almost 6 months early (3/19/2020 PDUFA date).
  6. The FDA approved diroximel fumarate (Vumerity, Biogen/Alkermes) on 10/30/2019 for the treatment of relapsing forms of multiple sclerosis. Diroximel fumarate is an oral prodrug of monomethyl fumarate, which allows the 505(b)(2) pathway based on Biogen’s dimethyl fumarate (Tecfidera) to be used. Biogen is licensing diroximel fumarate from Alkermes.
  7. The FDA approved luspatercept (Reblozyl, Celgene, Acceleron) on 11/8/2019 for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions.
  8. The FDA approved cefiderocol (Fetroja, Shionogi) on 11/15/2019 for the treatment of adult patients with complicated urinary tract infections (cUTI), including kidney infections caused by susceptible Gram-negative microorganisms, who have limited or no alternative treatment options. The package insert for cefiderocol will include a warning regarding higher all-cause mortality rate observed in cefiderocol-treated patients compared to those treated with other antibiotics in a trial in critically ill patients with multidrug-resistant Gram-negative bacterial infections.
  9. The FDA approved crizanlizumab (Adakveo, Novartis) on 11/15/2019 to reduce the frequency of vaso-occlusive crisis in patients aged 16 years or older with sickle cell disease. WAC for crizanlizumab is $2,357 per vial. With an average dose of three to four vials per month, WAC would be $7,071, or $9,428, per month.
  10. The FDA approved zanubrutinib (Brukinsa, BeiGene) on 11/13/2019 for the treatment of adults with mantle cell lymphoma who have received at least one prior therapy. WAC for a 30-day supply of zanubrutinib has been set at $12,935.
  11. The FDA approved givosiran (Givlaari, Alnylam) on 11/20/2019 for the treatment of adults with acute hepatic porphyria. WAC for givosiran is $39,000 per vial with an estimated annual cost after discount of $442,000.
  12. The FDA approved cenobamate (Xcopri, SK Life Science) on 11/21/2019 for the treatment of partial-onset seizures in adult patients with epilepsy. The FDA reports the most common ADR in the trials were somnolence, dizziness, fatigue, and diplopia. Cenobamate may cause a reduction in the QT interval > 20 milliseconds, so the FDA advises avoiding the drug in patients with Familial Short QT syndrome.
  13. The FDA approved voxelotor (Oxbryta, Global Blood Therapeutics) on 11/25/2019 for the treatment of sickle cell disease in patients 12 years and older. The drug had a PDUFA Date of 2/26/2020 and the FDA gave voxelotor Orphan Drug, Fast Track and Breakthrough Therapy Priority Designations.

ICER’s Review of Migraine Medications

ICER released a draft review of lasmiditan, ubrogepant and rimegepant in November 2019. ICER found the drugs to be comparable to each other in efficacy, but not as efficacious as triptans. Lasmiditan, ubrogepant and rimegepant would provide a benefit for patients with cardiovascular disease that have a contraindication to triptans, were not helped by triptans or do not tolerate them. Since triptans are available as generics, ICER concludes that triptans would provide a greater benefit at a lower cost than lasmiditan, ubrogepant or rimegepant. To reach a threshold of $150,000 per quality-adjusted life year ICER estimated an annual cost of $1,850 for lasmiditan, $1,780 for rimegepant and $1,740 for ubrogepant.

Weekly Update for the Week Ending November 30, 2019

12/3/2019

 
Regulatory Update
 
The FDA approved voxelotor (Oxbryta, Global Blood Therapeutics) on 11/25/2019 for the treatment of sickle cell disease in patients 12 years and older. The drug had a PDUFA Date of 2/26/2020 and the FDA gave voxelotor Orphan Drug, Fast Track and Breakthrough Therapy Priority Designations.
 
The FDA granted a priority review for risdiplam (RG7916) targeting spinal muscular atrophy and set a PDUFA date for 5/24/2020. It is the only drug in our knowledge base targeting spinal muscular atrophy.
 
The FDA assigned pemigatinib a Priority Review for the treatment of locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements and assigned a PDUFA date of 5/30/2020.
 
CymaBay has terminated clinical trials evaluating seladelpar as a treatment for non-alcoholic steatohepatitis (NASH) and primary sclerosing cholangitis (PSC) and put primary biliary cholangitis (PBC) studies on hold while it investigates atypical liver histological findings. There are eleven other investigational drugs in the Pharmaceutical Pipeline Tracker targeting NASH. 
 
Announced Research Updates
 
ChemoCentryx announced that in the 52-week, 331 patient, Phase III, ADVOCATE trial, 72.3% of patients treated with avacopan achieved remission [Birmingham Vasculitis Activity Score of zero and no glucocorticoid treatment for anti-neutrophil cytoplasmic antibody-associated (ANCA) vasculitis for at least the preceding four weeks] at 26 weeks compared to 70.1% with corticosteroids. Additionally, 65.7% of avacopan patients remained in remission at 52 weeks compared to 54.9% with glucocorticoids.
 
Published Research Updates
 
In a 141 patient, Phase IIb, open-label trial, eryaspase plus either gemcitabine or FOLFOX treated patients had an overall survival (OS) of 6.2 months compared to 4.9 months with standard chemotherapy and progression-free survival (PFS) of 2 months compared to 1.8 months in patients with advanced pancreatic adenocarcinoma with low asparagine synthetase (ASNS) expression. In the overall population OS was 6 months compared to 4.4 months and PFS was 2 months compared to 1.6 months. 
 
AstraZeneca/Lilly’s lanabecestat 104-week, 2,218 patient, Phase II/III, AMARANTH trial and the 78-week, 1,722 patient, Phase III DAYBREAK-ALZ trial were both terminated early after an interim analysis failed to demonstrate an improvement or slowing in decline in the Alzheimer Disease Assessment Scale–cognitive subscale (ADAS-Cog13) compared to placebo in patients with early or mild Alzheimer’s disease.
 
In the 107-week, Phase III SAkuraSky trial, of 83 patients with neuromyelitis optica spectrum disorder (NMOSD) that were receiving immunosuppressive therapy, 80% of patients treated with satralizumab were relapse free compared to 57% of those given placebo.
 
In the 681 patient, Phase II/III PALM Trial, mortality was 33.5% after treatment with REGN-EB3, 35.1% after MAb114, 53.1% with remdesivir compared to 49.7% with porgaviximab (ZMapp) in patients infected with the Ebola virus.
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