Perspectives
Specialty Pipeline Quarterly Update: SEPTEMBER 2022
Critical updates in an ever changing environment
September 30, 2022This quarterly pipeline wrap-up provides a review of newly approved specialty drugs, recent specialty drug launches, new indications and news of note on specialty drugs in the approval process. See separate articles for pipeline information on traditional drugs, biosimilars and cell/gene therapy.
New Drug Information
- Xenpozyme® (olipudase alfa-rpcp): The U.S. Food and Drug Administration (FDA) has approved Sanofi’s Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease type A and type B) in adult and pediatric patients. ASMD is a rare, progressive, and potentially life-threatening disease that results from a deficient activity of the enzyme acid sphingomyelinase (ASM), which is found in special compartments within cells called lysosomes and is required to breakdown lipids called sphingomyelin. If ASM is absent or not functioning as it should, sphingomyelin cannot be metabolized properly and accumulates within cells, eventually causing cell death and the malfunction of major organ systems. The deficiency of the lysosomal enzyme ASM is due to disease-causing variants in the sphingomyelin phosphodiesterase 1 gene (SMPD1). The estimated prevalence of ASMD is approximately 2,000 patients in the United States, Europe, and Japan.1 Xenpozyme was approved based on the Phase 3 ASCEND trial that demonstrated significantly improved lung function and significantly reduced spleen volume for Xenpozyme-treated patients at 52 weeks. Additionally, Xenpozyme’s data remained consistent throughout the 6.5-year outcomes for five adult patients with ASMD and two-year outcomes in 20 pediatric patients, as well as the open-label extension from the Phase 3 ASCEND trial in adults. Results showed the percent predicted lung function (DLco) mean increase was 55.3%, spleen volume mean decrease was 59.5%, and liver volume mean decrease was 43.7% compared to baseline of original study.1 Xenpozyme launch and price are pending.
- Stimufend® (pegfilgrastim-fpgk): Fresenius Kabi’s Stimufend was approved by the FDA as a pegfilgrastim biosimilar. Stimufend is indicated for use in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia. Stimufend is the fifth approved pegfilgrastim biosimilar using Amgen’s Neulasta as a reference product. The company expects to launch the product in a prefilled syringe early next year and in an on-body injector following FDA approval with pricing to follow.2
- Sotyktu® (deucravacitinib): The FDA approved BMS’ Sotyktu for the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy. Sotyktu is a first-in-class, oral, selective, allosteric tyrosine kinase 2 (TYK2) inhibitor. Sotyktu’s approval was based on the Phase 3 POETYK PSO-1 and POETYK PSO-2 trials that evaluated the percentage of patients who achieved Psoriasis Area and Severity Index (PASI) 75 and the percentage of patients who achieved static Physician’s Global Assessment (sPGA) score of 0 to 1 with at least a two-grade improvement from baseline, compared with placebo, at week 16.
- Findings from the POETYK PSO-1 and POETYK PSO-2 trials, respectively, included the following:
- Patients achieving PASI 75 response at week 16: 58% and 53% for deucravacitinib vs 13% and 9% for placebo (both P <.0001) and 35% (P<.0001) and 40% (P =.0004) for apremilast.3
- Patients achieving PASI 90 response at week 16: 36% and 27% for deucravacitinib vs 4% and 3% for placebo (both P <.0001) and 20% (P=.0002) and 18% (P =.0046) for apremilast.3
- Patients achieving sPGA 0/1 at week 16: 54% and 50% for deucravacitinib vs 7% and 9% for placebo (both P <.0001) and 32% and 34% for apremilast (both P <.0001).3
- Findings from the POETYK PSO-1 and POETYK PSO-2 trials, respectively, included the following:
Sotyktu has launched with an average wholesale price of $7,397 per bottle of 30 tablets.
- Rolvedon™ (eflapegrastim-xnst): The FDA approved Spectrum Pharmaceuticals’ Rolvedon injection to decrease the incidence of infection, as manifested by febrile neutropenia, in adult patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia. Rolvedon’s approval was based on two identical Phase 3 clinical trials, ADVANCE and RECOVER, which evaluated the safety and efficacy of Rolvedon in early-stage breast cancer patients for the management of neutropenia due to myelosuppressive chemotherapy. In both studies, Rolvedon demonstrated non-inferiority (NI) in mean duration of severe neutropenia (DSN) and a similar safety profile to pegfilgrastim.4 Rolvedon also demonstrated non-inferiority to pegfilgrastim in the mean DSN across all four cycles in both trials. Rolvedon is scheduled to launch fourth quarter 2022 with pricing to follow.
- Terlivaz™ (terlipressin): The FDA approved Terlivaz for injection. Terlivaz is the first FDA-approved product indicated to improve kidney function in adults with hepatorenal syndrome (HRS) with rapid reduction in kidney function, an acute and life-threatening condition requiring hospitalization. Terlivaz was approved based on the Phase 3, CONFIRM trial which used a surrogate endpoint of renal function improvement, avoidance of dialysis and short-term survival to demonstrate that Terlivaz reversed HRS-1 in 29% of patients who were treated with Terlivaz compared to 16% treated with placebo.5 In July 2020, the FDA Advisory Committee voted 8 to 7 to recommend approval of Terlivaz. The panel suggested that the benefits of Terlivaz outweigh the safety risks, Terlivaz and placebo groups experienced serious side effects at roughly the same rate: 65% versus 61%. Terlivaz launch and price are pending.
New Indications
- Pemazyre® (pemigatinib): The FDA expanded Incyte’s Pemazyre indication to include treatment of relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement.
September News
- “Bluebird bio has reported its financial results and business highlights for the second quarter of 2022, in which the company revealed the anticipated launch of both betibeglogene autotemcel (beti-cel) and elivaldogene autotemcel (eli-cel) gene therapies in the fourth quarter of the year. The news comes after both therapies were endorsed by the FDA Cell Tissue & Gene Therapy Advisory Committee (CTGTAC) in June 2022. Beti-cel is under priority review for the treatment of people with transfusion-dependent beta-thalassaemia (beta-thal), a rare genetic blood disease caused by a gene defect that impairs the ability of red blood cells to produce hemoglobin. Patients with the most severe form of beta-thal develop life-threatening anemia and have to undergo regular blood transfusions, a lengthy process typically needed every two to five weeks. Eli-cel is under priority review for the treatment of early active cerebral adrenoleukodystrophy (CALD) in patients under the age of 18 who do not have an available and willing matched sibling donor. CALD is a rare neurodegenerative disease that primarily affects young children and leads to irreversible loss of neurologic function and death. Beti-cel and eli-cel have Prescription Drug User Act Fee (PDUFA) goal dates of 19 August 2022 and 16 September 2022, respectively. If approved, the company anticipates that both therapies will be available in the fourth quarter of 2022.”6
- “Amylyx Pharmaceuticals Inc. shares climbed after its controversial treatment for amyotrophic lateral sclerosis won the support of a panel of U.S. regulatory advisers, putting the product on track for likely clearance. During a rare second panel meeting on Wednesday, the FDA peripheral and central nervous system drugs advisory committee voted 7-2 to back approval of the treatment. During a meeting in March, FDA advisers found that data from Amylyx’s relatively small initial trial was insufficient to prove that AMX0035 slowed ALS progression. The negative decision put the FDA in a bind, as the agency, under pressure from disease activists, has promised to exercise flexibility in approving drugs for dire illnesses such as ALS, given the urgent need for better treatments. New data from Amylyx “is not unequivocally persuasive, but nonetheless is quite reassuring,” said Liana Apostolova of Indiana University School of Medicine, who voted in favor of the drug this time after opposing it in March. She said she took into account that ALS “is a horrific disorder, it is a death sentence.””7
- “Apellis Pharmaceuticals, Inc., a global biopharmaceutical company and leader in complement, today announced top-line data at 24 months showing increased effects over time with intravitreal pegcetacoplan, an investigational, targeted C3 therapy, in the Phase 3 DERBY and OAKS studies in geographic atrophy (GA) secondary to age-related macular degeneration (AMD). In a pre-specified analysis of GA lesion growth over 24 months, both monthly and every-other-month (EOM) pegcetacoplan showed a clinically meaningful reduction in GA lesion growth from baseline compared to sham (all p-values are nominal): DERBY: 19% monthly, p=0.0004; 16% EOM, p=0.0030. OAKS: 22% monthly, p<0.0001; 18% EOM, p=0.0002. Between months 18-24, the pegcetacoplan treatment effect accelerated compared to previous six-month periods, with robust reductions of GA lesion growth versus sham (all p-values are nominal). The increased effects were driven by a greater slowing of lesion growth by pegcetacoplan and not by an increase in the lesion growth rate in the sham group, which was highly consistent over each of the four six-month intervals (1.0+/-0.05 mm2).”8
SPECIALTY NEW PRODUCT APROVALS IN THE PAST TWELVE MONTHS
Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Administration | Month Approved | |
terlipressin | Terlivaz® | Mallinckrodt | Improve kidney function in adults with hepatorenal syndrome (HRS) | SC | September 2022 | |
deucravacitinib | Sotyktu® | Bristol Myers Squibb | Plaque psoriasis | Oral | September 2022 | |
spesolimab-sbzo | Spevigo® | Boehringer Ingelheim | Generalized pustular psoriasis (GPP) flares | IV | September 2022 | |
olipudase alfa-rpcp | Xenpozyme® | Sanofi | Acid sphingomyelinase deficiency | IV | August 2022 | |
FT218 (sodium oxybate, controlled release) | Lumryz™
Tentative approval |
Avadel Pharmaceuticals | Narcolepsy | Oral | July 2022 | |
vutrisiran | Amvuttra® | Alnylam Pharmaceuticals | Polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis | SC | June 2022 | |
edaravone, oral | Radicava ORS® | Mitsubishi Tanabe Pharma | Amyotrophic lateral sclerosis (ALS) | Oral | May 2022 | |
trientine tetrahydrochloride | Cuvrior™ | Orphalan | Wilson’s disease | Oral | May 2022 | |
treprostinil dry powder for inhalation | Tyvaso DPI® | United Therapeutics Corp | New dry powder formulation of for treatment of pulmonary arterial hypertension | Inhaled | May 2022 | |
mavacamten | Camzyos® | Bristol-Myers Squibb | Symptomatic obstructive hypertrophic cardiomyopathy (oHCM) | Oral | April 2022 | |
ganaxolone | Ztalmy™ | Marinus Pharmaceuticals, Inc. | Seizures associated with CDKL5 deficiency disorder | Oral | March 2022 | |
mitapivat | Pyrukynd® | Agios Pharmaceuticals | Pyruvate kinase (PK) deficiency | Oral | February 2022 | |
sutimlimab-jome | Enjaymo® | Sanofi US | Cold agglutinin disease (CAD) | IV | February 2022 | |
faricimab-svoa | Vabysmo® | Genentech | Diabetic macular edema | Intravitreal | January 2022 | |
abrocitinib | Cibinqo® | Pfizer And Lilly | Atopic dermatitis | Oral | January 2022 | |
inclisiran | Leqvio® | Novartis | Hyperlipidemia | SC | January 2022 | |
tezepelumab | Tezspire® | AstraZeneca and Amgen | Asthma across phenotypes | SC | January 2022 | |
tralokinumab-ldrm | Adbry® | Leo Pharma Inc | Atopic dermatitis | SC | December 2021 | |
levoketoconazole | Recorlev® | Strongbridge Biopharma | Cushing’s syndrome | Oral | December 2021 | |
efgartigimod alfa-fcab | Vyvgart® | Argenx | Generalized myasthenia gravis | IV | December 2021 | |
budesonide | Tarpeyo® | Calliditas Therapeutics | Primary IgA nephropathy (IgAN) | Oral | December 2021 | |
vosoritide | Voxzogo® | BioMarin | Achondroplasia | SC | November 2021 | |
ropeginterferon alfa-2b-njft | Besremi® | PharmaEssentia | Polycythemia vera (PV) | SC | November 2021 | |
treprostinil inhalation powder | Yutrepia®
Tentative approval |
Liquidia | Pulmonary arterial hypertension | Inhaled dry powder | November 2021 | |
ranibizumab via ocular implant | Susvimo® | Genentech | Age-related macular degeneration | Intravitreal injection | October 2021 | |
triamcinolone acetonide | Xipere® | Clearside Biomedical and Bausch + Lomb (Bausch Health) | Macular edema associated with uveitis | Suprachoroidal injection | October 2021 | |
avacopan | Tavneos® | Chemocentryx | ANCA-associated vasculitis | Oral | October 2021 | |
allogeneic processed thymus tissue-agdc | Rethymic® | Enzyvant (Sumitomo Dainippon, Sumitovant) | Certain forms of congenital athymia | Surgical | October 2021 |
NEW INDICATIONS FOR APPROVED SPECIALTY PRODUCTS
Generic Name | Brand Name | Manu-facturer | New Indication(s) | Date Approved | |
ustekinumab | Stelara® | Johnson & Johnson (Janssen) | Pediatric patients aged 6 years and older with psoriatic arthritis (PsA) | August 2022 | |
belimumab | Benlysta® | GlaxoSmithKline | Patients aged 5 to 17 years old with active lupus nephritis | July 2022 | |
ruxolitinib topical cream | Opzelura® | Incyte | Nonsegmental vitiligo in adults and adolescents 12 years of age and older | July 2022 | |
pegloticase + methotrexate | Krystexxa® + methotrexate | Horizon | Co-administration with methotrexate for the treatment of chronic gout | July 2022 | |
setmelanotide | Imcivree® | Rhythm Pharmaceuticals | Obesity and control of hunger in adult and pediatric patients 6 years of age and older with Bardet-Biedl syndrome (BBS) | June 2022 | |
dupilumab | Dupixent® | Sanofi and Regeneron | For add-on maintenance treatment of pediatric patients aged six months to fve years old with moderate to severe atopic dermatitis | June 2022 | |
baricitinib | Olumiant® | Lilly and Incyte | Alopecia areata | June 2022 | |
mycophenolate mofetil | Cellcept® | Roche | Prophylaxis of organ rejection to pediatric recipients of allogenic heart and liver transplants | June 2022 | |
brolucizumab-dbll | Beovu® | Novartis | Diabetic macular edema | June 2022 | |
risdiplam | Evrysdi® | Genentech | Pre-symptomatic babies under two months of age with Spinal Muscular Atrophy (SMA) | May 2022 | |
dupilumab | Dupixent® | Sanofi Pharmaceuticals | To treat eosinophilic esophagitis (EoE) in adults and pediatric patients 12 years and older weighing at least 40 kilograms (which is about 88 pounds) | May 2022 | |
baricitinib | Olumiant® | Lilly and Incyte | For the treatment of COVID-19 in hospitalized adults requiring supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO) | May 2022 | |
upadacitinib | Rinvoq® | Abbvie | Adults with active ankylosing spondylitis | April 2022 | |
ravulizumab-cwvz | Ultomiris® | AstraZeneca (Alexion) | Generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor antibody-positive | April 2022 | |
remdesivir | Veklury® | Gilead | Certain pediatric patients 28 days of age and older weighing at least three kilograms with positive results of direct SARS-CoV-2 viral testing | April 2022 | |
upadacitinib | Rinvoq® | Abbvie | Moderately to severely active ulcerative colitis | March 2022 | |
tocilizumab | Actemra® IV | Genentech | Adults with giant cell arteritis | February 2022 | |
von Willebrand factor, recombinant | Vonvendi® | Takeda | Bleeding episodes in adults with von Willebrand disease (VWD) | January 2022 | |
risankizumab-rzaa | Skyrizi® | Abbvie | Treatment of adults with active psoriatic arthritis | January 2022 | |
upadacitinib | Rinvoq® | Abbvie | Adults and adolescents with moderate to severe atopic dermatitis (eczema) | January 2022 | |
remdesivir | Veklury® | Gilead | Mild to moderate COVID-19 in adults and adolescents who are at high risk for progression to severe COVID-19, including hospitalization or death | January 2022 | |
secukinumab | Cosentyx® | Novartis | Active enthesitis-related arthritis in four years and older, and active juvenile psoriatic arthritis (JPsA) in patients two years and older | December 2021 | |
apremilast | Otezla® | Amgen | Mild to moderate plaque psoriasis who are candidates for phototherapy or systemic therapy | December 2021 | |
voxelotor | Oxbryta® | Global Blood Therapeutics, Inc | Pediatric indication and new dispersible tablet for oral suspension dosage form of the sickle hemoglobin polymerization inhibitor for treatment of sickle cell disease (SCD) in patients aged four to 11 years | December 2021 | |
abatacept | Orencia® | BMS | Prophylaxis of acute graft versus host disease (aGVHD) in combination | December 2021 | |
tofacitinib | Xeljanz® | Pfizer | Ankylosing spondylitis (AS) | December 2021 | |
upadacitinib | Rinvoq® | Abbvie | Psoriatic arthritis | December 2021 | |
dupilumab | Dupixent® | Sanofi and Regeneron | Add-on treatment of pediatric patients aged 6 to 11 years of age with uncontrolled moderate-to-severe asthma | October 2021 | |
bictegravir, emtricitabine and tenofovir alafenamide | Biktarvy® | Gilead | Expanded pediatric indication for the integrase strand transfer inhibitor (INSTI)-based single-tablet regimen and new low-dose tablet formulation for treatment of HIV-1 infected pediatric patients weighing at least 14 kg | October 2021 |
ONCOLOGY PRODUCT APROVALS IN THE PAST TWELVE MONTHS
Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Administration | Date Approved | |
eflapegrastim-xnst | Rolvedon® | Spectrum Pharmaceuticals and Hanmi Pharmaceutical | Chemotherapy-induced neutropenia | SC | September 2022 | |
bortezomib | Bortezomib® | Hospira | Multiple myeloma
mantle cell lymphoma |
IV or SC | May 2022 | |
alpelisib | Vijoice® |
Novartis
|
Severe manifestations of PIK3CA-related overgrowth Spectrum (PROS) | Oral | April 2022 | |
lutetium Lu 177 vipivotide tetraxetan | Pluvicto® | Novartis | Prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) | IV | March 2022 | |
sirolimus topical gel | Hyftor® | Nobelpharma | Facial angiofibroma | Gel | March 2022 | |
relatlimab and nivolumab | Opdualag® | Bristol Myers Squibb | Certain forms of metastatic melanoma | IV | March 2022 | |
pacritinib | Vonjo® | CTI BioPharma | Myelofibrosis patients with severe thrombocytopenia | Oral | February 2022 | |
tebentafusp-tebn | Kimmtrak® | Immunocore | Metastatic uveal melanoma (mUM) | IV | January 2022 | |
cabazitaxel | N/A | Accord | Metastatic castration-resistant prostate cancer | IV | December 2021 | |
sirolimus albumin-bound nanoparticles for injectable suspension | Fyarro® | Aadi Bioscience | Metastatic or locally advanced malignant perivascular epithelioid cell tumor (PEComa) | IV | November 2021 | |
asciminib | Scemblix® | Novartis | Certain forms of chronic myeloid leukemia (CML) | Oral | October 2021 |
NEW INDICATIONS FOR APPROVED ONCOLOGY DRUGS
Generic Name | Brand Name | Manufacturer | New Indication | Date Approved |
pemigatinib | Pemazyre® | Incyte | R/R myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement | August 2022 |
capmatinib | Tabrecta® | Novartis | Certain forms of metastatic non-small cell lung cancer (NSCLC) | August 2022 |
darolutamide | Nubeqa® | Bayer and Orion | In combination with docetaxel for treatment of metastatic hormone-sensitive prostate cancer (mHSPC) | August 2022 |
fam-trastuzumab deruxtecan-nxki | Enhertu® | Daiichi Sankyo and AstraZeneca | Unresectable or metastatic HER2-low breast cancer | August 2022 |
crizotinib | Xalkori® | Pfizer | Inflammatory myofibroblastic tumor (IMT) | July 2022 |
carfilzomib | Kyprolis® | Onyx (Amgen) | R/R multiple myeloma (RRMM) who have received one to three lines of therapy. | June 2022 |
lisocabtagene maraleucel | Breyanzi® | Bristol-Myers Squibb | R/R large B-cell lymphoma (LBCL) after failure of first-line therapy | June 2022 |
dabrafenib and trametinib | Tafinlar® and Mekinist® | Novartis | Adult and pediatric patients aged six years and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment | June 2022 |
fam-trastuzumab deruxtecan-nxki | Enhertu® | Daiichi Sankyo and AstraZeneca | Unresectable or metastatic HER2-positive breast cancer who have received a prior anti-HER2-based regimen | May 2022 |
tisagenlecleucel | Kymriah® | Novartis | R/R follicular lymphoma (FL) after two prior lines of treatment | May 2022 |
nivolumab and ipilimumab | Opdivo® and Yervoy® | Bristol Myers Squibb | Uresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) | May 2022 |
ivosidenib | Tibsovo® | Agios Pharmaceuticals | In combination with azacitidine for newly diagnosed acute myeloid leukemia (AML) with a susceptible IDH1 mutation, as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy | May 2022 |
azacitadine | Vidaza® | Bristol Myers Squibb (Celgene) | New indication for the nucleoside metabolic inhibitor for treatment of pediatric patients aged one month and older with newly diagnosed juvenile myelomonocytic leukemia (JMML) | May 2022 |
axicabtagene ciloleucel | Yescarta® | Kite | Second line treatment of adults with R/R large B-cell lymphoma | April 2022 |
pembrolizumab | Keytruda® | Merck | Advanced endometrial carcinoma that is microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation | March 2022 |
abemaciclib | Verzenio® | Lilly | In combination with endocrine therapy (tamoxifen or aromatase inhibitor) for adjuvant treatment of adults with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) | October 2021 |
brexucabtagene autoleucel | Tecartus® | Gilead Sciences | Adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) | October 2021 |
BIOSIMILAR PRODUCT APROVALS IN THE PAST TWELVE MONTHS
Generic Name | Brand Name | Reference
Product |
Manufacturer | Indication(s) | Route of Administration | Month Approved |
pegfilgrastim-fpgk | Stimufend® | Neulasta® biosimilar | Fresenius Kabi | Neutropenia | IV or SC | September 2022 |
adalimumab-bwwd 100mg/mL | Hadlima® HC | Humira® biosimilar | Samsung Bioepis and Organon | Autoimmune | SC | August 2022 |
ranibizumab-eqrn First interchangeable biosimilar for Lucentis | Cimerli® | Lucentis® biosimilar | Coheres biosciences/bioeq | Age-related macular degeneration | Injection into the eye | August 2022 |
pegfilgrastim-pbbk | Fylnetra™ | Neulasta® | Amneal and Kashiv (Adello) | Neutropenia | IV or SC | May 2022 |
bevacizumab-maly | Alymsys™ | Avastin® | mAbxience, Insud Pharma; Amneal | Oncology | IV | April 2022 |
filgrastim-ayow | Releuko™ | Amneal | Neutropenia | Blood modifying | IV | February 2022 |
insulin glargine-aglr | Rezvoglar™ | Lantus® | Eli Lilly | Diabetes | SC | December 2021 |
adalimumab-aqvh | Yusimry™ | Humira® | Coherus | Autoimmune | SC | December 2021 |
ranibizumab-nuna | Byooviz™ | Lucentis® | Samsung Bioepis | Age-related macular degeneration | Intravitreal | September 2021 |
insulin glargine-yfgn
First interchangeable biosimilar for Lantus |
Semglee™ | Lantus® | Mylan and Biocon | Diabetes | SC | July 2021 |
SPECIALTY PIPELINE
Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Administration | Anticipated Approval date* | |
eflornithine and sulindac | Flynpovi® | Cancer Prevention Pharmaceuticals | Familial adenomatous polyposis (FAP) | Oral | Delayed | |
donislecel purified allogeneic deceased donor pancreas derived islets of Langerhans | Lantidra® | CellTrans | Brittle type 1 diabetes | IV | Delayed | |
SH-111 | N/A | Shorla Pharma | Pediatric patients with T-cell leukemia | IV | Delayed | |
inolimomab | Leukotac® | Farmaceutici Spa | Steroid-refractory acute graft-versus-host disease | IV | Delayed | |
efbemalenograstim alfa | Ryzneuta™ | Evive Biotech | Chemotherapy-induced neutropenia | SC | Delayed | |
penpulimab | Annik™ | Akeso and Sino Biopharmaceutical | Metastatic nasopharyngeal carcinoma | IV | Delayed | |
tislelizumab | N/A | BeiGene and Novartis | Certain forms of esophageal squamous cell carcinoma (ESCC) | IV | Delayed | |
miglustat (for AT-GAA combination with cipaglucosidase alfa) | N/A | Amicus Therapeutics | Pompe disease | Oral | Delayed | |
teclistamab | N/A | Johnson & Johnson (Janssen) | Relapsed or refractory multiple myeloma | IV or SC | Delayed | |
sodium phenylbutyrate and taurursodiol | Albrioza™ | Amylyx Pharmaceuticals, Inc. | Amyotrophic lateral sclerosis (ALS) | Oral | September 2022 | |
sodium thiosulfate, STS | Pedmark™ | Fennec Pharmaceuticals | Prevention of ototoxicity induced by cisplatin | IV | September 2022 | |
futibatinib | N/A | Taiho Oncology | Metastatic cholangiocarcinoma | Oral | September 2022 | |
subcutaneous furosemide, sc2Wear furosemide infusor-drug/device combination | Furoscix™ | ScPharmaceuticals | Diuresis in patients with worsening heart failure | SC minipump | October 2022 | |
tremelimumab | N/A | AstraZeneca | Certain forms of unresectable hepatocellular carcinoma | IV | October 2022 | |
cipaglucosidase alfa (in combination with miglustat) | N/A | Amicus Therapeutics | Pompe disease | IV | October 2022 | |
SPN-830 (apomorphine infusion pump) | N/A | Supernus Pharmaceuticals | Parkinson’s disease | SC Minipump | October 2022 | |
teplizumab | Tzield™ | Provention Bio | Diabetes | IV | November 2022 | |
sparsentan | N/A | Travere Therapeutics | IgA nephropathy (IgAN) | Oral | November 2022 | |
poziotinib | N/A | Spectrum Pharmaceuticals, Inc. | Certain forms of metastatic non-small cell lung cancer (NSCLC) | Oral | November 2022 | |
mirvetuximab soravtansine | N/A | ImmunoGen | Certain forms of folate receptor alpha-high platinum-resistant ovarian cancer | IV | November 2022 | |
131I-omburtamab | Omblastys™ | Y-mAbs | Pediatric patients with central nervous system and leptomeningeal metastasis from neuroblastoma | IV | November 2022 | |
RBX2660 fecal microbiota transplant | Rebyota™ | Ferring (Rebiotix) | Clostridioides difficile | Fecal microbiome transplant | November 2022 | |
pegcetacoplan | N/A | Apellis Pharmaceuticals | Geographic atrophy secondary to AMD | Intravitreal | November 2022 | |
toripalimab | Tuoyi™ | Junshi Biosciences and Coherus | In combination with gemcitabine and cisplatin for first-line treatment for patients with advanced recurrent or metastatic nasopharyngeal carcinoma; monotherapy for second-line or later treatment of recurrent or metastatic NPC patients with disease progression on or after platinum-containing chemotherapy | IV | December 2022 | |
adagrasib | N/A | Mirati Therapeutics | Second-line treatment of advanced non-small cell lung cancer (NSCLC) | Oral | December 2022 | |
palovarotene | N/A | Ipsen (Clementia) | Fibrodysplasia ossificans progressiva (FOP) | Oral | December 2022 | |
mosunetuzumab | N/A | Genentech (Roche) | R/R follicular lymphoma (FL) | IV | December 2022 | |
NexoBrid | N/A | MediWound | Burn tissue | Gel | January 2023 | |
ACER-001 (sodium phenylbutyrate) | N/A | Acer Therapeutics and Relief Therapeutics | Urea cycle disorders | Oral | January 2023 | |
tofersen | N/A | Biogen | Superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (SOD1-ALS | Intrathecal injection | January 2023 | |
lecanemab | N/A | Eisai | Alzheimer’s disease | IV | January 2023 | |
olutasidenib | N/A | Forma and Rigel | Relapsed or refractory acute myeloid leukemia | Oral | February 2023 | |
elacestrant | N/A | Radius Health and Menarini | ER+/ HER2- advanced or metastatic breast cancer | Oral | February 2023 | |
omidubicel | N/A | Gamida Cell | Blood cancer | Cell therapy | February 2023 | |
omaveloxolone | Omav™ | Reata Pharmaceuticals, Inc. | Friedreich’s ataxia | Oral | February 2023 | |
efanesoctocog alfa | N/A | Sanofi and Sobi | Hemophilia A | IV | February 2023 | |
velmanase alfa | N/A | Chiesi Global Rare Diseases | Alpha-mannosidosis | IV | March 2023 | |
[Vic-] trastuzumab duocarmazine | N/A | Byondis | HER2-positive unresectable locally advanced or metastatic breast cancer | IV | May 2023 | |
nogapendekin alfa + BCG | Anktiva™ | ImmunityBio | Bladder cancer | IV | May 2023 | |
momelotinib | N/A | Sierra Oncology, Inc. | Myelofibrosis | Oral | June 2023 | |
ritlecitinib | N/A | Pfizer | Alopecia areata | Oral | June 2023 |
* Anticipated approval dates are predictions made by Prime Therapeutics based on industry information.
BIOSIMILAR PIPELINE
Generic name | Brand name | Manufacturer | Indication(s) | Route of administration | Anticipated approval Date* | |
Oncology | ||||||
MYL-1402O | N/A (Avastin® biosimilar) | Mylan NV and Biocon Ltd. | Oncology | IV | 2022 | |
SB8 | N/A (Avastin® biosimilar) | Samsung Bioepis and Merck | Oncology | IV | 2022 | |
FKB238 | N/A (Avastin® biosimilar) | Centus Biotherapeutics | Oncology | IV | 2022 | |
BAT-1706 | N/A (Avastin® biosimilar) | Bio-thera solutions | Oncology | IV | 2022 | |
CT-P16 | N/A (Avastin® biosimilar) | Celltrion | Oncology | IV | 2022 | |
EG12014 | N/A (Herceptin® biosimilar) | Sandoz | Oncology | IV | 4Q2022 | |
TX05 | N/A (Herceptin® biosimilar) | Tanvex | Oncology | IV | 2022 | |
Blood Modifiers | ||||||
lupifil-p | N/A (Neulasta® biosimilar) | Lupin | Neutropenia | SC | 2022 | |
lapelga | N/A (Neulasta® biosimilar) | Apotex/Accord | Neutropenia | SC | 2022 | |
TX01 | N/A (Neupogen® biosimilar) | Tanvex BioPharma | Neutropenia | SC | 2022 | |
grastofil | N/A (Neupogen® biosimilar) | Apotex/Accord | Neutropenia | SC | 2022 | |
Ophthalmology | ||||||
xlucane | N/A (Lucentis® biosimilar) | Xbrane & Bausch | Age-related macular degeneration | Injection into the eye | 2023 | |
MYL-1701P | N/A (Eylea® biosimilar) | Mylan | Age-related macular degeneration | Injection into the eye | 2022 | |
Autoimmune | ||||||
CT-P17 | Yuflyma® (Humira® biosimilar) | Celltrion | Autoimmune | SC | 2022 | |
MSB11022 | Idacio® (Humira® biosimilar) | Fresenius | Autoimmune | SC | 2022 | |
MSB11456 | N/A (Actemra® IV/SC biosimilar) | Fresenius | Autoimmune | IV/SC | 2Q2023 |
REFERENCES
- https://www.sanofi.com/en/media-room/press-releases/2022/2022-06-28-05-30-00-2469974
- https://www.fresenius-kabi.com/news/fresenius-kabi-receives-us-fda-approval-for-biosimilar
- https://www.empr.com/home/news/fda-approves-sotyktu-for-moderate-to-severe-plaque-psoriasis/
- https://www.businesswire.com/news/home/20220909005522/en/Spectrum-Pharmaceuticals-Receives-FDA-Approval-for-ROLVEDON%E2%84%A2-eflapegrastim-xnst-Injection
- https://www.prnewswire.com/news-releases/mallinckrodt-receives-us-fda-approval-for-terlivaz-terlipressin-for-injection-for-the-treatment-of-hepatorenal-syndrome-hrs-301624894.html
- https://www.pmlive.com/pharma_news/bluebird_bio_reveals_plans_to_launch_two_gene_therapies_1453120
- https://www.bloomberg.com/news/articles/2022-09-07/lou-gehrig-s-disease-drug-from-amylyx-gets-rare-second-fda-meeting?leadSource=uverify%20wall
- https://www.globenewswire.com/news-release/2022/08/24/2503714/0/en/Apellis-Announces-24-Month-Results-Showing-Increased-Effects-Over-Time-with-Pegcetacoplan-in-Phase-3-DERBY-and-OAKS-Studies-in-Geographic-Atrophy-GA.html
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