Perspectives
Specialty Pipeline Update: March 2022
March 29, 2022New drugs, new indications and news of note
This monthly 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 article for pipeline information on traditional drugs.
New Drug Information
Pyrukynd®(mitapivat): U.S. Food and Drug Administration (FDA) has approved Pyrukynd (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare, debilitating, lifelong hemolytic anemia. Pyrukynd is a first-in-class, oral PK activator and the first approved disease-modifying therapy for this disease. The Phase 3 ACTIVATE trial of Pyrukynd achieved its primary endpoint by demonstrating a statistically significant increase in hemoglobin in patients with PK deficiency who are not regularly transfused with 40% of patients randomized achieving a hemoglobin response, compared to 0 patients randomized to placebo. The Phase 3 ACTIVATE-T trial of Pyrukynd also achieved its primary endpoint of demonstrating a statistically significant and clinically meaningful reduction in transfusion burden for patients who are regularly transfused (33% of patients achieved a transfusion reduction response, compared with individual historical transfusion burden standardized to 24 weeks). Twenty two percent of patients were transfusion-free during the fixed-dose period.1 Pyrukynd has launched with an average wholesale price (AWP) of $552 per tablet.
Releuko® (filgrastim-ayow): The FDA approved Amneal’s Releuko as a biosimilar version of Amgen’s Neupogen® (filgrastim). Releuko is indicated to:
- Decrease the incidence of infection‚ as manifested by febrile neutropenia‚ in patients with nonmyeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a significant incidence of severe neutropenia with fever
- Reduce the time to neutrophil recovery and the duration of fever, following induction or consolidation chemotherapy treatment of patients with acute myeloid leukemia (AML)
- Reduce the duration of neutropenia and neutropenia-related clinical sequelae‚ febrile neutropenia, in patients with nonmyeloid malignancies undergoing myeloablative chemotherapy followed by bone marrow transplantation (BMT)
- Reduce the incidence and duration of sequelae of severe neutropenia‚ in symptomatic patients with congenital neutropenia‚ cyclic neutropenia‚ or idiopathic neutropenia
Releuko was approved on two clinical trials that confirmed pharmacokinetic and bioavailability between Releuko and Neupogen. Releuko has not been granted interchangeable status by the FDA. The FDA noted that biosimilars marketed in the U.S. typically have launched with initial list prices 15% to 35% lower than comparative list prices of the reference products. According to IQVIA®, U.S. annual sales for filgrastim for the 12 months ended December 2021 were $407 million, of which $275 million represents biosimilar sales.2 Releuko is the third biosimilar to Neupogen to be approved by the FDA after Sandoz’s Zarxio® (filgrastim-sndz) and Pfizer’s Nivestym® (filgrastim-aafi). Releuko has launched with AWP $273.60 – $437.76 per vial/syringe.
Vonjo® (pacritinib): The FDA granted accelerated approval of CTI BioPharma’s Vonjo for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. Vonjo is an oral kinase inhibitor with specificity for JAK2 and IRAK1, without inhibiting JAK1. Vonjo was approved based on the Phase 3 PERSIST-2 trial which demonstrated that 29% of patients with baseline platelet counts below 50 × 109/L who were treated with pacritinib 200mg had a reduction in spleen volume of at least 35% compared to 3% of patients receiving best available therapy, which included ruxolitinib.3The recommended dosage of Vonjo is 200mg orally twice daily. Vonjo has launched with a wholesale acquisition cost (WAC) of $19,500 per month.
Carvykti® (ciltacabtagene autoleucel; cilta-cel): The FDA approved Legend Biotech Corporation’s Carvykti for the treatment of adults with relapsed or refractory multiple myeloma (RRMM) who have received four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. Carvykti is a chimeric antigen receptor T-cell (CAR-T) therapy with two B-cell maturation antigen (BCMA)-targeting single domain antibodies and is given as a one-time infusion with a recommended dose range of 0.5 to 1.0 x 106CAR-positive viable T cells per kg of body weight. In the pivotal CARTITUDE-1 study, deep and durable responses were seen in patients with RRMM, with a high overall response rate (ORR) of 98 percent including 78 percent of the patients achieving stringent complete response (sCR).4 At a median of 18 months follow-up, the median duration of response (DOR) was 21.8 months. Carvykti has launched with a wholesale acquisition cost (WAC) of $465,000.
New Indications
None
March News
“The FDA is staying cautious after data came out suggesting that one of BioMarin’s experimental gene therapies might have the potential to drive cancers. Although details remain sparse, BioMarin said that its gene therapy for the rare disease phenylketonuria, or PKU, will remain on hold after the FDA asked for additional non-clinical — i.e., lab-based or animal studies — to assess “the theoretical oncogenic risk to human study participants.” Those studies, the company said, are “expected to take several quarters.””5
“The adoption of biosimilars tends to accelerate quickly after market introduction, according to Cardinal Health. And their use has grown significantly since 2015. Momentum is “stronger than ever” in the biosimilar space, Cardinal Health’s specialty solutions president Heidi Hunter said. Last year, Viatris’ Semglee became the first interchangeable biosimilar, meaning it can be substituted for the original insulins without a doctor’s input. The drug references Sanfoi’s long-acting insulin, Lantus. Upon launching in November, Viatris said the list price would be set at about $400 for a package of five 3 mL pens, which is about $20 cheaper than the list price for the branded version. The company also simultaneously launched an unbranded version of its insulin glargine product at a 65% list price discount, which the company called “the lowest WAC for any insulin glargine pen on the market to date.” While it’s unclear what 2022 will hold in store for biosimilar pricing and coverage, Cardinal says one message is clear: Momentum in this space is accelerating. Analysts say biosimilars are on track to reduce U.S. drug expenditure by $133 billion by 2025. “2022 is set to be a turning point in the U.S., as biosimilars expand into new therapeutic areas and sites of care, and reimbursement models continue to evolve,” Hunter said.”6
“With two of its prized gene therapies on clinical hold and another, Zynteglo, pulled from the market in Europe seven months ago after it was approved for use, these are not the best of times for bluebird bio. So after updating the status of lovo-cel, eli-cel and beti-cel (Zynteglo) in its fourth quarter and 2021 earnings report, bluebird illuminatedits dire financial situation, saying that its cash needs “raise substantial doubt regarding its ability to continue as a going concern.”7
“Sales of Bristol Myers Squibb’s Revlimid crescendoed at $12.8 billion in 2021, the final year of exclusivity for the multiple myeloma treatment. But now, BMS can no longer hold off generic competition. Last month, Sandoz and Stada Arzneimittel revealed that launches of their generic versions of Revlimid are underway in Europe. Now Teva has become the first to do the same in the U.S. Teva will provide Lenalidomide capsules in 5mg, 10mg, 15mg and 25mg strengths, it said, for the treatment of multiple myeloma in combination with dexamethasone, certain myelodysplastic syndromes and mantle cell lymphoma following specific prior treatment. The company did not note what it would charge.”8
SPECIALTY NEW PRODUCT APPROVALS IN THE PAST TWELVE MONTHS
Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Admin | Month Approved |
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 |
maralixibat | Livmarli® | Mirum Pharmaceuticals | Alagille syndrome (ALGS) | Oral | September 2021 |
ruxolitinib cream | Opzelura® | Incyte Corporation | Atopic dermatitis | Topical | September 2021 |
lonapegsomatropin | Skytrofa® | Acendis | Pediatric growth hormone deficiency(GHD) | SC | September 2021 |
avalglucosidase alfa | Nexviazyme® | Sanofi-Aventis | Pompe disease | IV | August 2021 |
anifrolumab-fnia | Saphnelo® | AstraZeneca | Systemic lupus erythematosus (SLE) | IV | August 2021 |
selexipag | Uptravi® | Johnson & Johnson | Pulmonary arterial hypertension | IV | July 2021 |
odevixibat | Bylvay® | Albireo | Progressive familial intrahepatic cholestasis | Oral | July 2021 |
belumosudil | Rezurock® | Kadmon | Chronic graft-versus-host disease (cGVHD) | Oral | July 2021 |
allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen-dsat | StrataGraft™ | Mallinckrodt | Deep partial-thickness thermal burns | Topical | June 2021 |
sofosbuvir/velpatasvir | Epclusa® pellets | Gilead | Hepatitis C | Oral | June 2021 |
aducanumab-avwa | Aduhelm® | Biogen | Alzheimer’s disease | IV | June 2021 |
plasminogen (human-tvmh) | Ryplazim® | Prometic Life Sciences Inc | Plasminogen deficiency type 1 | IV | June 2021 |
pegcetacoplan | Empaveli® | Apellis | Paroxysmal nocturnal hemoglobinuria | SC | May 2021 |
NEW INDICATIONS FOR APPROVED SPECIALTY PRODUCTS
Generic Name | Brand Name | Manufacturer | New Indication(s) | Date Approved |
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 |
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 4 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 |
ruxolitinib | Jakafi® | Incyte | Steroid-refractory chronic graft versus host disease (SR chronic GVHD) in adults and pediatric patients 12 years and older | September 2021 |
calcium, magnesium, potassium and sodium oxybates | Xywav® | Jazz Pharmaceuticals | Idiopathic hypersomnia | August 2021 |
mepolizumab | Nucala® | Glaxo Smith Kline | Chronic rhinosinusitis with nasal polyps | July 2021 |
tacrolimus | Prograf® | Astellas Pharma | Prevent organ rejection in adult and pediatric patients receiving lung transplantation | July 2021 |
ravulizumab-cwvz | Ultomiris® | Alexion | Pediatric patients with paroxysmal nocturnal hemoglobinuria (PNH) | June 2021 |
elexacaftor, tezacaftor, and ivacaftor | Trikafta® | Vertex Pharmaceuticals | Ages 6 to 11 with at least one F508del mutation in the CFTR gene (the gene defective in CF) or a CFTR mutation that is responsive to the therapy | June 2021 |
ozanimod | Zeposia® | Bristol-Myers Squibb | Treatment of adults with moderately to severely active ulcerative colitis (UC) | May 2021 |
omalizumab | Xolair® | Genentech | New prefilled syringe for treatment of persistent asthma and chronic idiopathic urticaria | April 2021 |
risankizumab-rzaa | Skyrizi® | AbbVie | New autoinjector and a prefilled syringe with needle stock prevention for treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy | April 2021 |
ONCOLOGY PRODUCT APPROVALS IN THE PAST TWELVE MONTHS
Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Admin | Date Approved |
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 |
tisotumab vedotin-tftv |
Tivdak® | Seagen | Certain forms of cervical cancer | IV | September 2021 |
mobocertinib (TAK-788) | Exkivity® | Takeda Pharmaceuticals | Metastatic non-small cell lung cancer (mNSCLC) | Oral | September 2021 |
belzutifan | Welireg® | Merck | Von Hippel-Lindau (VHL) disease | Oral | August 2021 |
dostarlimab-gxly | Jemperli™ | GlaxoSmithKline (Tesaro) and AnaptysBio | Certain forms of solid tumors | IV | August 2021 |
asparaginase erwinia chrysanthemi-rywn | Rylaze® | Jazz Pharmaceuticals | Acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) | IV | June 2021 |
sotorasib | Lumakras® | Amgen | Certain forms of lung cancer | Oral | May 2021 |
infigratinib | Truseltiq® | QED Therapeutics (BridgeBio) | Certain forms of cholangiocarcinoma | Oral | May 2021 |
leuprolide mesylate 50 mg depot | Camcevi® 42mg | Foresee Pharmaceuticals | Palliative treatment of advanced prostate cancer | Injectable suspension | May 2021 |
amivantamab-vmjw | Rybrevant™ | Johnson & Johnson | Certain forms of non-small cell lung cancer | IV | May 2021 |
dostarlimab-gxly | Jemperli™ | GlaxoSmithKline (Tesaro) and AnaptysBio | Endometrial cancer | IV | April 2021 |
loncastuximab tesirine (lonca) | Zynlonta™ | ADC Therapeutics | R/R diffuse large B-cell lymphoma (DLBCL) | IV | April 2021 |
NEW INDICATIONS FOR APPROVED ONCOLOGY DRUGS
Generic Name | Brand Name | Manufacturer | New Indication | Date Approved |
rituximab | Rituxan® | Genentech | B-cell lymphoma, Burkitt lymphoma, Burkitt-like lymphoma, or mature B-cell acute leukemia | December 2021 |
pembrolizumab | Keytruda® | Merck | Stage IIB or IIC melanoma | December 2021 |
pembrolizumab | Keytruda® | Merck | Adjuvant treatment of renal cell carcinoma (RCC) | November 2021 |
atezolizumab | Tecentriq® | Roche (Genentech) | Stage II-IIIA non-small cell lung cancer (NSCLC) | October 2021 |
pembrolizumab | Keytruda® | Merck | Combination with chemotherapy, with or without bevacizumab, treatment of patients with persistent, recurrent or metastatic cervical cancer whose tumors express PD-L1 | October 2021 |
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 |
evolocumab | Repatha® | Amgen | Add-on treatment to diet alone or together with certain other therapies for patients aged 10 years and older with heterozygous familial hypercholesterolemia (HeFH) and homozygous familial hypercholesterolemia (HoFH) | September 2021 |
cabozantinib | Cabometyx® | Exelixis and Ipsen | Patients aged 12 and older with locally advanced or metastatic differentiated thyroid cancer (DTC) that has progressed following prior vascular endothelial growth factor receptor (VEGFR)-targeted therapy and who are radioactive iodine-refractory or ineligible | September 2021 |
cetuximab | Erbitux® | Lilly | Certain forms of metastatic colorectal cancer (CRC) with a BRAF V600E mutation | September 2021 |
ivosidenib | Tibsovo® | Agios Pharmaceuticals | Locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation, which is a rare type of cancer in the bile ducts in the liver | September 2021 |
zanubrutinib | Brukinsa® | Beigene USA | Marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based therapy | September 2021 |
zanubrutinib | Brukinsa® | Beigene USA | Rare indolent B-cell lymphoma Waldenstrom’s Macroglobulinemia (WM) | August 2021 |
nivolumab | Opdivo® | Bristol-Myers Squibb | Certain forms of urothelial carcinoma (UC) | August 2021 |
pembrolizumab | Keytruda® | Merck | First-line treatment of patients with RCC | August 2021 |
pembrolizumab | Keytruda® | Merck | Certain forms of triple negative breast cancer (TNBC) in combination with chemotherapy | July 2021 |
pembrolizumab and lenvatinib | Keytruda® and Lenvima® | Merck and Eisai | Certain forms of advanced endometrial carcinoma that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) | July 2021 |
enfortumab vedotin-ejfy | Padcev® | Astellas and Seagen | Treatment of patients with locally advanced or metastatic urothelial cancer who have received a PD-1/L1 inhibitor and are ineligible for cisplatin | July 2021 |
daratumumab and hyaluronidase-fihj | Darzalex Faspro® | Janssen Biotech and Genmab | Combination regimen with pomalidomide and dexamethasone (d-Pd) for treatment of patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy | July 2021 |
avapritinib | Ayvakit® | Blueprint Medicines Corp | Adults with advanced systemic mastocytosis (SM) | June 2021 |
pembrolizumab | Keytruda® | Merck | Certain combination for first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma | May 2021 |
nivolumab | Opdivo® | Bristol-Myers Squibb | Adjuvant treatment of patients with resected esophageal or gastroesophageal junction (GEJ) cancer after neoadjuvant chemoradiation therapy (CRT) | May 2021 |
nivolumab | Opdivo® | Bristol-Myers Squibb | Certain forms of advanced or metastatic gastric cancer, gastroesophageal junction cancer (GEJC) or esophageal adenocarcinoma (EAC) | May 2021 |
lorlatinib | Lorbrena® | Pfizer | Treatment of previously untreated advanced ALK-positive non-small cell lung cancer (NSCLC) | April 2021 |
sacituzumab govitecan-hziy | Trodelvy® | Gilead Sciences | Triple-negative breast cancer that has spread to other parts of the body | April 2021 |
nivolumab | Opdivo® | Bristol-Myers Squibb | Initial treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer and esophageal adenocarcinoma | April 2021 |
BIOSIMILAR PRODUCT APPROVALS IN THE PAST TWELVE MONTHS
Generic Name | Brand Name | Reference
Product |
Manufacturer | Indication(s) | Route of Admin | Month Approved |
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® biosimilar | Coherus | Autoimmune | SC | December 2021 |
ranibizumab-nuna | Byooviz™ | Lucentis® biosimilar | 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 Admin | Anticipated Approval date* |
CPP-1X/sul (eflornithine and sulindac) | N/A | 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 |
FT218 (sodium oxybate, controlled release) | N/A | Avadel Pharmaceuticals | Narcolepsy | Oral | Delayed |
bimekizumab | Bimzelx® | UCB Pharma | Psoriasis | SC | Delayed |
inolimomab | Leukotac® | Farmaceutici Spa | Steroid-refractory acute graft-versus-host disease | IV | Delayed |
penpulimab (AK105) | N/A | Akeso and Sino Biopharmaceutical | Metastatic nasopharyngeal carcinoma | IV | Delayed |
sintilimab | N/A | Innovent Biologics and Lilly | Nonsquamous non-small cell lung cancer (NSCLC) | IV | March 2022 |
relatlimab and nivolumab | N/A | Bristol Myers Squibb | Certain forms of metastatic melanoma | IV | March 2022 |
ganaxolone | Ztalmy™ | Marinus Pharmaceuticals, Inc. | Seizures associated with CDKL5 deficiency disorder (CDD) | Oral | March 2022 |
vadadustat | Vafseo™ | Akebia Therapeutics | Anemia due to chronic kidney disease (CKD) | Oral | March 2022 |
efbemalenograstim alfa | Ryzneuta™ | Evive Biotech | Chemotherapy-induced neutropenia | SC | March 2022 |
toripalimab | Tyoyi™ | Junshi Biosciences and Coherus | Certain forms of recurrent or metastatic nasopharyngeal carcinoma | IV | April 2022 |
vutrisiran | N/A | Alnylam Pharmaceuticals | Polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis | SC | April 2022 |
mavacamten | N/A | Bristol-Myers Squibb | Symptomatic obstructive hypertrophic cardiomyopathy (oHCM) | Oral | April 2022 |
surufatinib | N/A | Hutchmed | Pancreatic and extra-pancreatic neuroendocrine tumor | Oral | April 2022 |
parsaclisib | N/A | Incyte | Certain forms of mantle cell lymphoma (MCL) | Oral | April 2022 |
edaravone, oral | N/A | Mitsubishi Tanabe Pharma | Amyotrophic lateral sclerosis (ALS) | Oral | May 2022 |
trientine tetrahydrochloride | Cuprior™ | Orphalan | Wilson’s disease | Oral | May 2022 |
tapinarof | N/A | Dermavant (Roivant) | Plaque psoriasis | Topical | May 2022 |
miglustat (for AT-GAA combination with cipaglucosidase alfa) | N/A | Amicus Therapeutics | Pompe disease | Oral | May 2022 |
ublituximab | N/A | TG Therapeutics | Certain forms of chronic lymphocytic leukemia (CLL) | IV | June 2022 |
spesolimab | N/A | Boehringer Ingelheim | Generalized pustular psoriasis (GPP) flares | IV | June 2022 |
sodium phenylbutyrate and taurursodiol | N/A | Amylyx Pharmaceuticals, Inc. | Amyotrophic lateral sclerosis (ALS) | Oral | June 2022 |
sodium phenylbutyrate | N/A | Acer Therapeutics and Relief Therapeutics | Urea cycle disorders (UCDs) | Oral | June 2022 |
tislelizumab | N/A | BeiGene and Novartis | Certain forms of esophageal squamous cell carcinoma (ESCC) | Injection | July 2022 |
roflumilast cream (ARQ-151) | N/A | Arcutis Biotherapeutics, Inc. | Plaque psoriasis | Topical | July 2022 |
cipaglucosidase alfa* (in combination with miglustat) | N/A | Amicus Therapeutics | Pompe disease | IV | July 2022 |
olipudase alfa | N/A | Sanofi (Genzyme) | Niemann-Pick disease | IV | July 2022 |
parsaclisib | N/A | Incyte | Certain forms of follicular lymphoma (FL) | Oral | August 2022 |
teplizumab | Tzield™ | Provention Bio | Diabetes | IV | August 2022 |
deucravacitinib | N/A | Bristol Myers Squibb | Plaque psoriasis | Oral | September 2022 |
ublituximab | N/A | TG Therapeutics | Relapsing multiple sclerosis | IV | September 2022 |
poziotinib | N/A | Spectrum Pharmaceuticals, Inc. | Certain forms of metastatic non-small cell lung cancer (NSCLC) | Oral | November 2022 |
adagrasib | N/A | Mirati Therapeutics | Second-line treatment of advanced non-small cell lung cancer (NSCLC) | Oral | December 2022 |
* Anticipated approval dates are predictions made by Prime Therapeutics based on industry information.
BIOSIMILAR PIPELINE
Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Admin | 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 |
BEVZ92 | N/A (Avastin® biosimilar) | mAbxience, Insud Pharma; Amneal | Oncology | IV | 2022 |
CT-P16 | N/A (Avastin® biosimilar) | Celltrion | Oncology | IV | 2022 |
EG12014 | N/A (Herceptin® biosimilar) | Sandoz | Oncology | IV | 2022 |
TX05 | N/A (Herceptin® biosimilar) | Tanvex | Oncology | IV | 2022 |
Blood Modifiers | |||||
MSB11455 | Stimufend® (Neulasta® biosimilar) | Fresenius Kabi | Neutropenia | IV or SC | 2022 |
TPI-120 | N/A (Neulasta® biosimilar) | Amneal and Kashiv (Adello) | Neutropenia | IV or SC | 2022 |
lupifil-p | N/A (Neulasta® biosimilar) | Lupin | Neutropenia | SC | 2022 |
lapelga | N/A (Neulasta® biosimilar) | Apotex/Accord | Neutropenia | SC | 2022 |
TX-01 | N/A (Neupogen® biosimilar) | Tanvex BioPharma | Neutropenia | SC | 2022 |
grastofil | N/A (Neupogen® biosimilar) | Apotex/Accord | Neutropenia | SC | 2022 |
Ophthalmology | |||||
FYB201 | Cimerli® (Lucentis® biosimilar) | Coheres biosciences/bioeq | Age-related macular degeneration | Injection into the eye | 2022 |
MYL-1701P | N/A (Eylea® biosimilar) | Mylan | Age-related macular degeneration | Injection into the eye | 2022 |
Autoimmune | |||||
AVT02 | N/A (Humira® biosimilar) | Alvotech | Autoimmune | SC | 2022 |
CT-P17 | Yuflyma® (Humira® biosimilar) | Celltrion | Autoimmune | SC | 2022 |
adalimumab-bwwd (SB5 100mg/mL) | N/A (Humira® biosimilar) | Samsung Bioepis and Organon | Autoimmune | SC | 2022 |
FDA APPROVED GENE/CELL THERAPY PRODUCT APPROVALS
Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Admin | Month Approved |
ciltacabtagene autoleucel (cilta-cel) | Carvykti™ | Johnson & Johnson (Janssen) | R/R multiple myeloma | CAR-T | February 2022 |
allogeneic processed thymus tissue adgc | Rethymic™ | Enzyvant | Congenital athymia | Surgical | October 2021 |
idecabtagene vicleucel (bb2121, ide-cel) | Abecma™ | Bristol-Myers Squibb and bluebird bio | Multiple myeloma | CAR-T | March 2021 |
lisocabtagene maraleucel
(liso-cel) |
Breyanzi™ | Bristol-Myers Squibb | Large B cell lymphoma (LBCL) | IV | February 2021 |
brexucabtagene autoleucel | Tecartus™ | Kite | Mantle cell lymphoma | CAR T-cell therapy | July 2020 |
onasemnogene abeparvovec-xioi | Zolgensma™ | AveXis | Spinal Muscular Atrophy | IV | May 2019 |
voretigene neparvovec-rzyl | Luxturna® | Spark Therapeutics | Biallelic RPE65 mutation-associated retinal dystrophy | Subretinal injection | December 2017 |
axicabtagene ciloleucel | Yescarta® | Kite | LBCL/follicular lymphoma (FL) | IV | October 2017 |
tisagenlecleucel | Kymriah® | Novartis | Acute lymphoblastic leukemia/LBCL | IV | August 2017 |
GENE/CELL THERAPY 2022 PIPELINE
Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Admin | Anticipated Approval date* |
betibeglogene autotemcel | N/A | Bluebird Bio | Transfusion-dependent beta-thalassemia (TDT) | IV | August 2022 |
elivaldogene autotemcel | Lenti-D™ | Bluebird Bio | Cerebral adrenoleukodystrophy (CALD) | IV | September 2022 |
etranacogene dezaparvovec | EtranaDez™ | CSL Behring | Hemophilia B | IV | 3Q2022 |
valoctocogene roxaparvovec | Roctavian™ | BioMarin | Hemophilia A | IV | 4Q2022 |
eladocagene exuparvovec | PTC-AADC | PTC Therapeutics | Aromatic L-amino acid decarboxylase (AADC) deficiency | Intracerebral | 4Q2022 |
References
- https://investor.agios.com/news-releases/news-release-details/agios-announces-fda-approval-pyrukyndr-mitapivat-first-disease
- https://www.businesswire.com/news/home/20220302005951/en/Kashiv-Biosciences-Receives-Approval-for-Its-First-Biosimilar-RELEUKOTM-filgrastim-ayow
- https://www.prnewswire.com/news-releases/cti-biopharma-announces-fda-accelerated-approval-of-vonjo-pacritinib-for-the-treatment-of-adult-patients-with-myelofibrosis-and-thrombocytopenia-301492159.html
- https://www.businesswire.com/news/home/20220228006246/en/CARVYKTI%E2%84%A2-ciltacabtagene-autoleucel-BCMA-Directed-CAR-T-Therapy-Receives-U.S.-FDA-Approval-for-the-Treatment-of-Adult-Patients-With-Relapsed-or-Refractory-Multiple-Myeloma
- https://endpts.com/biomarin-gene-therapy-could-be-on-hold-for-several-quarters-after-fda-mandates-new-preclinical-tests/
- https://endpts.com/last-year-was-one-of-the-most-eventful-in-us-biosimilars-history-but-these-analysts-say-2022-should-be-a-turning-point/
- https://www.fiercepharma.com/pharma/bluebird-bios-cash-needs-raise-doubts-about-its-ability-continue-going-concern
- https://www.fiercepharma.com/pharma/bristol-myers-squibbs-revlimid-finally-faces-competition-us-tevas-generic-launch?utm_source=email&utm_medium=email&utm_campaign=LS-NL-FiercePharma&oly_enc_id=6999D8336467C2P
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