Therapeutic DeliveryVol. 12, No. 9 Industry NewsFree AccessIndustry news update covering May 2021Louise Rosenmayr-TempletonLouise Rosenmayr-Templeton *Author for correspondence: E-mail Address: louise.templeton@towerpharmacon.comhttps://orcid.org/0000-0002-1315-8398Tower Pharma Consulting, Bellakreuzstrasse 21, Berndorf, 2560, AustriaSearch for more papers by this authorPublished Online:10 Aug 2021https://doi.org/10.4155/tde-2021-0047AboutSectionsPDF/EPUB ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareShare onFacebookTwitterLinkedInRedditEmail Keywords: competitor intelligenceemerging technologiesintellectual propertymonoclonal antibodiesparenteral deliverypartneringAcquisitionsJazz Pharmaceuticals closes out purchase of GW PharmaceuticalsEarly in the month, Jazz Pharmaceuticals plc (Dublin, Ireland) made public that it had completed its purchase of GW Pharmaceuticals, plc (Cambridge, UK) [1]. GW Pharmaceuticals has a proprietary cannabinoid platform from which one product has already reached the market in both the US and in Europe. Epidiolex® (cannabidiol) oral solution is licensed in the US to treat seizures resulting from Lennox-Gastaut syndrome, Dravet syndrome or tuberous sclerosis complex in patients of 1 year and older. The same product is marketed in Europe as Epidyolex® where it is used together with clobazam for the treatment of Lennox-Gastaut syndrome and Dravet syndrome associated seizures and also in tuberous sclerosis complex patients of 2 years and older. Its other marketed product is Sativex®, which is a botanical extract containing delta-9-tetrahydrocannibinol and cannabidiol as the main active principles, for the treatment of spasticity in multiple sclerosis. It has been granted marketing authorization in a number of countries and is currently being evaluated for the same indication in Phase III clinical studies in the US, where it is known as nabiximols. Other clinical phase candidates in GW Pharmaceuticals’ pipeline include those for the treatment of autism and schizophrenia [2].The acquisition, which was first announced in February 2021 [3], saw GW Pharmaceuticals valued at US$7.2 billion or $220.00 per American depositary share, $200.00 of which was in cash and the remainder in Jazz ordinary shares. Its purchase will bolster and expand Jazz’s existing neuroscience portfolio and add an additional revenue stream with respect to sales of cannabidiol oral solution, which has already achieved $510 million in revenue per year within the first 2 years of launch.Nexturn Bio invests in RosVivo’s mRNA technologyOn 26 May, Nexturn Bio Inc. (Yongin, South Korea) announced that it had invested around US$5.5 million in RosVivo Therapeutics Inc. (NV, USA) and taken a 50% share in the company [4]. The acquisition enables Nexturn and its South Korean parent, Nexturn Bioscience Co., Ltd, to enter the mRNA therapeutic arena. RosVivo is developing miRNA and siRNA therapies for the treatment of Type 2 diabetes and other related metabolic diseases [5]. The premise that these therapies will be effective is based on research which shows that the development of Type 2 diabetes is linked to the loss of specific miRNAs, in particular miR-10b-5p, and that their replacement can reverse the disease [6]. RosVivo’s lead candidate, RSVI-301, is a potential treatment for Type 2 diabetes and obesity and has been shown to be effective in human cell models and mice [5]. RosVivo’s approach differs from current marketed therapies, in that it targets disease suppression and not blood sugar control. In a separate line of research, the company is investigating the use of miRNAs to suppress SARS-CoV-2 proliferation and cytokine storm as a potential treatment for severely ill COVID-19 patients [5].The investment by Nexturn will help RosVivo progress its exciting but early-stage research into clinical trials.Licensing agreementsActuate & Lantern partner to speed development of 9-ING-41A few days into the month came the announcement that Lantern Pharma Inc. (TX, USA) would apply its RADR® artificial intelligence (AI) platform to the development of 9-ING-41 from Actuate Therapeutics Inc. (TX, USA) [7]. 9-ING-41 is a glycogen synthase kinase-3β (GSK-3β) inhibitor which is currently being assessed in Phase II trials for pancreatic cancer, myelofibrosis and other cancers [8].Lantern’s RADR technology applies machine learning and AI to genomic and clinical trial data to identify patient subgroups most likely to respond to oncology clinical candidates, to clarify mechanisms of action and to identify potential advantageous combinations of cancer drugs [9]. In turn, these data enable the stratification of patients for clinical studies based on validated biomarker signatures identified by AI. RADR currently contains more than 4.6+ billion data points from 140+ drug/tumor interactions and clinical data from thousands of patients. According to the company website, its use has been shown in numerous case studies to have greater than 80% accuracy under blinded conditions. Lantern applies the its RADR platform to support its own cancer drug pipeline and accelerate the development of its partnered projects through efficient design of clinical trials. In the case of the collaboration with Actuate, the goal is to model predictively tumor sensitivity to 9-ING-41 and identify biomarkers for patients who will respond.No financial details of the research and development agreement were made public. However, it is anticipated that work will begin straight away and that any intellectual property developed as a result will be co-owned by the two firms. It is understood that payment is in the form of upfront equity in Actuate subject to certain conditions of the collaboration being achieved. Lantern will receive further equity if the collaboration efforts are used in future development work [9].Lilly & MiNA collaborate on saRNAOn 11 May Eli Lilly (IN, USA) and MiNA Therapeutics Ltd (London, UK) issued a press release regarding their plan to collaborate on the discovery of new therapies using MiNA’s saRNA technology platform [10]. Small activating RNAs are double-stranded, noncoding RNA comprising 21 nucleotides with two-nucleotide overhangs at the end of each strand [11]. saRNA is transported into the cell nucleus from the cytosol in the form of a complex with the AGO2 protein. Once in the nucleus, it acts to upregulate transcription of the target gene.MiNA Therapeutics’ first clinical candidate from their platform, MTL-CEBPA, targets upregulation of the transcription factor CEBPA, which has been shown to be downregulated in liver and other cancers. Two Phase I clinical trials of this drug, formulated as a lipid nanoparticle, in patients with advanced liver cancer yielded positive safety data with promising indications of efficacy especially when combined with the current standard treatment, sorafenib; thus, demonstrating the potential of this approach [11,12].MiNA Therapeutics’ first clinical candidate from their platform, MTL-CEBPA, targets upregulation of the transcription factor CEBPA, which has been shown to be downregulated in liver and other cancers. Two Phase I clinical trials of this drug, formulated as a lipid nanoparticle, in patients with advanced liver cancer yielded positive safety data with promising indications of efficacy especially when combined with the current standard treatment, sorafenib; thus, demonstrating the potential of this approach [11,12].The collaboration with Lilly involves research into up to five targets chosen by Lilly and aligned with the company’s development focus. In return, MiNA will be renumerated with US$25 million upfront and be eligible for up to US$245 million pro target in milestone payments plus tiered royalties based on sales resulting from the collaboration. Lilly will conduct the preclinical and clinical programs and have sole rights to bring any products developed in the course of the collaboration to market.Coeptis & VyGen-Bio agree codevelopment options for VyGen-Bio technologiesCoeptis Pharmaceuticals, Inc. (PA, USA) made public on 18 May that it had signed two separate exclusive option agreements with VyGen-Bio, Inc. (FL, USA) with respect to the codevelopment of the Florida company’s technologies for CD38 related cancers [13]. CD38 associated cancers include multiple myeloma, chronic lymphocytic leukemia and acute myeloid leukemia. The technologies are derived from VyGen-Bio’s gene-edited antibody resistant natural killer (NK) (GEAR-NK) platform, which itself is based on research carried out at the Karolinska Institute in Sweden.The first agreement concerns CD38-GEAR-NK, which is an autologous NK cell therapy candidate with the potential to prevent the destruction of CD38+ NK cells by anti-CD38 monoclonal antibodies. Loss of CD38+ NK cells during anti-CD38 antibody therapy has been demonstrated both in vitro and in vivo [14,15]. CD38-GEAR-NK has the potential to overcome this issue by gene-editing NK cells removed from the patient. The cells are then re-administered to the patient together with anti-CD38 therapy. The gene-edited NK cells should then be protected from the effects of the antibody therapy.The second agreement relates to an in vitro diagnostic test, known as CD38 diagnostic. It is designed to identify oncology patients who would benefit from treatment with anti-CD38 antibody therapy.The financial details of the two codevelopment option agreements were not disclosed, but in both cases, Coeptis has made a nonrefundable payment and has until 31 December 2021 to exercise its rights under the agreements and purchase the codevelopment rights to CD38-GEAR-NK and/or CD38 diagnostic. If the CD38-GEAR-NK option is activated, multiple myeloma is foreseen as the first indication to be investigated.Genentech to collaborate with Pieris for new therapies for respiratory & ophthalmic indicationsOn 25 May, Pieris Pharmaceuticals Inc. (MA, USA) announced that it had signed a research collaboration and license agreement with Genentech (CA, USA; part of Roche) with the objective of developing new treatments for respiratory and ophthalmic disease based on the firm’s Anticalin® discovery platform [16]. The Anticalin technology is based on research into naturally occurring lipocalins. These are small ligand binding proteins responsible for transporting molecules such as vitamins and steroids in the body [17,18]. The lipocalins are around 160–180 amino acid residues in size and consist of a single polypeptide chain. Their tertiary structure consists of a conserved beta-barrel structure connected to four hypervariable loops. It is the composition of these loops that determines binding specificity. Anticalin proteins retain the overall structure of the lipocalins, but their affinity and specificity can be tuned by alteration of the loop region using Pieris’ Anticalin technology platform to enable their binding to specific small molecule or protein targets. They can also be manufactured as fusion products with other proteins or antibodies to achieve multitarget molecules [18].The agreement foresees Pieris receiving an initial payment from Genentech of US$20 million, followed by more than US$1.4 billion contingent on the company meeting specific milestones over multiple programs. Sales of marketed products arising from the program are subject to tiered royalties. During the collaboration, Pieris will lead the discovery and early preclinical research efforts while Genentech will be responsible for later key activities such as Chemistry, Manufacturing and Controls (CMC), clinical development and commercialization. The agreement also provides Genentech with the option to choose additional targets. This option is associated with an option exercise fee.Product approvalsFirst therapy for cerebral adrenoleukodystrophy takes one step closer to EU approvalAt their May meeting, the EMA’s Committee for Medicinal Products for Human Use granted a positive opinion on Skysona™ (elivaldogene autotemcel), a gene therapy for use in children suffering from cerebral adrenoleukodystrophy (CALD) [19–21]. This inherited condition is caused by defects in the ABCD1 gene, which carries the genetic information for the translation of the adrenoleukodystrophy protein. The protein is responsible for the metabolism of very long chain fatty acids in the body and, without it, they accumulate in cells over time particularly in the adrenal gland and white matter of the brain and spinal cord. This accumulation results in inflammatory reactions and damage to the myelin sheath surrounding the nerves which is critical to their proper functioning. The incidence of genetic abnormalities in the X-linked ABCD1 gene is one in 21,000, with the disease affecting mainly boys. About 40% of those suffering from adrenoleukodystrophy will go on to develop the severe cerebral variant of the disease. CALD has a very poor prognosis with around 50% of patients dying within 5 years of symptom onset (typically between 3 and 12 years). To date, stem cell therapy is the only treatment option for children suffering from CALD.Skysona is indicated for the one-time treatment of early cerebral adrenoleukodystrophy in children of less than 18 years who have a ABCD1 mutation and no access to a leukocyte antigen-matched sibling hematopoietic stem cell donor. The product was developed by Bluebird Bio (MA, USA) [20,21]. It consists of immature autologous bone marrow cells which have been genetically altered using a lentivirus vector carrying the gene ABCD1 for the ALDP protein. On infusion the altered cells are distributed throughout the body where they differentiate into various healthy cell types including brain cells capable of producing the ALDP protein. The protein is then available to metabolize the build-up of VLFA, alleviate symptoms and hinder further neurological decline. The EMA based its opinion on the outcome of a Phase II/III clinical study involving 32 boys suffering from CALD aged 17 years or less. Twenty-four months following treatment, 30 patients were evaluated, 27 of whom met the trial’s primary end-point of survival with no major functional disability. The results from the study were also evaluated against one involving 59 patients who had received a stem cell transplantation. The most serious side effect reported was low blood cell levels. Enrolment in a second Phase III trial is currently underway and all patients from the initial Phase II/III study are being monitored as part of a long-term study. Approval for the product by the European Commission is anticipated mid-2021.Rybrevant™ approval targets lung cancer with EGFR Exon 20 insertion mutationsRybrevant (amivantamab-vmjw), a bispecific human G1-type antibody specifically for the treatment of locally advanced or metastatic non-small-cell lung cancer (NSCLC) patients identified as having EGFR exon 20 insertion mutations, was approved by the US FDA on 21 May [22,23]. The therapy was developed by Janssen Pharmaceutical Companies (PA, USA) (part of Johnson and Johnson) in collaboration with Genmab A/S (Copenhagen, Denmark) using Genmab’s DuoBody® technology platform [24]. The approval is not only a first for the targeted treatment of NSCLC sufferers with exon 20 insertion mutations, but also for a bispecific antibody discovered using Genmab’s technology. Rybrevant is approved as a second-line treatment for adults whose cancer has progressed despite chemotherapeutic intervention with platinum-based drugs. A companion diagnostic, Guardant360® CDx liquid biopsy blood test, from Guardian Health (CA, USA), was approved concurrently by the US regulatory authority.EGFR exon 20 insertion mutations are the third most common activating EGFR mutation. Their presence results in poorer prognosis and lower median overall survival rates (16.2 months) even compared with other types of EGFR mutation in NSCLC. Amivantamab-vmjw acts to inhibit tumor growth by binding to EGFR and MET receptors on the tumor cell surface. This binding ultimately leads to death of the cancer cells through a number of mechanisms.Rybrevant is presented as a 50 mg/ml solution in a single use vial containing 350 mg of active ingredient. The injection is diluted with 5% dextrose or 0.9% sodium chloride prior to intravenous (iv.) infusion and dosed weekly for 4 weeks (initial dose spilt over 2 days). The dosing frequency is then decreased to 2 weeks and continued until either toxicity becomes unacceptable or the cancer progresses. The dose is 1050 mg or 1400 mg based on body weight at the onset of treatment, with two dose reductions to up to 50% of the dose foreseen in the event of nontolerability.Until Rybrevant’s approval there was no targeted treatment for NSCLC associated with EGFR exon 20 insertion mutations. This was granted on the basis of overall response rate (ORR) and duration of response (DOR) from the Phase I CHRYSALIS study, which evaluated amivantamab-vmjw monotherapy in patients who had previously received platinium-based chemotherapy as part of the first study cohort. This study is ongoing and will also investigate the bispecific antibody in combination with other anticancer drugs.With respect to monotherapy, 81 patients suffering locally advanced or metastatic NSCLC with the target mutation type were administered Rybrevant based on body weight, as described previously. The ORR in patients previously administered platinum-based chemotherapy was 40% (95% CI: 29–51), split between 3.7% achieving complete response and 36% having a partial response as measured by the Response Evaluation Criteria in Solid Tumors Version 1.1 when assessed by Blinded Independent Central Review. The average DOR was 11.1 months and 63% of patients had a DOR of ≥6 months.Adverse effects which occurred in 20% or more of patients included rash, infusion related reactions, paronychia, muscle and bone pain, shortness of breath, nausea, fatigue, edema, stomatitis, cough, constipation and vomiting. In 11% of patients, serious or severe adverse reactions such as pneumonia, infusion related reactions, pneumonitis/interstitial lung disease, dyspnea, pleural effusion and rash resulted in complete withdrawal of treatment, while in 78%, dosing was temporarily stopped due to side effects.Clinical trials are ongoing and continuing US approval of Rybrevant may depend on confirmatory clinical results. Janssen has already applied for regulatory approval of Rybrevant in a number of territories including Europe.Sotorasib approval offers further hope for NSCLC patientsOn 28 May the FDA granted accelerated approval for Lumakras™ (sotorasib), which is indicated for the treatment of KRAS G12C-mutated locally advanced or metastatic NSCLC in adults who have previously received at least one other anticancer therapy [25–27]. Lumakras is marketed by Amgen (CA, USA) and is dosed orally once daily at a recommended dose of 960 mg (8 × 120 mg tablets) until the cancer progresses or toxicity becomes prohibitive. If severe side effects occur, the dose can be reduced twice to a minimum of 240 mg daily.The KRAS G12C mutation is identified in approximately 13% of US patients with nonsquamous NSCLC. The prognosis for patients with this mutation is very poor, with the average progression-free survival time following second line therapy being only 4 months. Sotorasib’s mechanism of action is the formation of a permanent covalent bond with a cysteine group of KRASG12C. Binding inactivates the protein and stops the downstream signaling which promotes tumor growth. The wild-type KRAS is unaffected [25,26].In order for the drug to be prescribed, the presence of the KRAS G12C mutation must be confirmed in plasma or in the tumor using an FDA approved biomarker diagnostic. Amgen collaborated with Guardant Health (CA, USA) on the development of the plasma test, while the tumor-based diagnostic was worked up in conjunction with Qiagen (Hilden, Germany). These tests, namely the Guardant360® CDx (plasma) and Qiagen therascreen® KRAS RGQ PCR kit (tissue), have also been approved by the FDA [27].The basis of sotorasib’s approval was ORR and DOR data from a group of 124 patients taking part in the CodeBreaK 100 clinical trial. These patients had previously received first-line therapy in the form of immunotherapy and/or chemotherapy, but had suffered further disease progression. Data analysis showed that therapy with Lumakras gave an ORR of 36% (95% CI: 28–45), where ORR was defined as ≥30% tumor shrinkage. In addition, 81% (95% CI: 73–87) of the 124 participants studied achieved disease control, in other words, a complete response, partial response or stable disease for longer than 3 months. Treatment had, on average, a DOR of 10 months at the time of the analysis. The most typical side effects (≥20%) were diarrhea, muscle/skeletal pain, nausea, fatigue, hepatotoxicity and cough. In 9% of patients, the severity of the side effects was sufficiently significant to result in Lumakras administration being permanently stopped. Two of the most serious adverse effects are hepatotoxicity, which in some cases resulted in dose reduction or discontinuation of treatment and more rarely interstitial lung disease/pneumonitis, which can be fatal.Studies on sotorasib are still ongoing, and its efficacy is currently being evaluated for the treatment of 13 other tumor varieties bearing the KRAS G12C mutation in world-wide clinical programs. Interestingly, one of the FDA’s postmarking conditions of approval is a trial to investigate if similar clinical outcomes can be achieved at a lower dose. Amgen have filed for approval for Lumakras in a number of different jurisdictions including the EU, the UK and Australia.Clinical trialsREGEN-COV™ effective in reducing risk of serious illness in nonhospitalized COVID-19 patientsIn a breaking news session at this year’s American Thoracic Society International Conference, Regeneron Pharmaceuticals Inc. (NY, USA) presented key data from a Phase III pivotal study into the efficacy and safety of their proprietary antibody combination REGEN–COV (casirivimab with imdevimab) for the treatment of COVID-19 [28]. These antibodies target the receptor binding domain of the SARS-CoV-2 viral spike protein. In vitro binding studies involving virus-like particles with surface-bound spike protein variants suggest that REGEN–COV is also active against current variants of SARS-CoV-2 [29].The randomized, double-blind, placebo-controlled study involved 4567 outpatients suffering from COVID-19. The participants were all at risk of developing severe COVID-19 symptoms due to their age (over 50 years old) and/or underlying health conditions. These conditions included asthma, other chronic lung disease, obesity and cardiovascular disease. The study showed that, compared with placebo administration, either 1200 or 2400 mg REGEN–COV resulted in a 70% (p = 0.0024) and 71% (p < 0.0001) reduction, respectively, in hospitalization or death up to day 29. In addition, COVID-19 symptoms disappeared faster (median of 10 days vs 14 with placebo) with both doses. Viral load at 7 days was significantly decreased compared with placebo (0.71 log10 copies/ml with 1200 mg and 0.86 log10 copies/ml with 2400 mg REGEN–COV). An evaluation of REGEN–COV safety at day 169 showed that serious adverse reactions could be mainly attributed to COVID-19 itself, with 1.1, 1.3 and 4% occurring in the cohorts dosed with 1200 mg REGEN–COV, 2400 mg REGEN–COV and placebo respectively. REGEN–COV associated side effects were mainly infusion reaction related and hypersensitivity. One death occurred in each of the REGEN–COV dosed groups, and four in the placebo group.REGEN–COV is currently authorized in the USA for emergency use in adults and children of 12 years and over weighing at least 40 kg who are at high-risk of severe disease or hospitalization [29]. The criteria for use are confirmation of SARS-CoV-2 infection by direct viral testing and mild-to-moderate COVID-19 disease. Administration should take place within 10 days of symptom onset by iv. infusion or subcutaneous (sc.) injection. It is not authorized for those requiring in-patient treatment for COVID-19 or oxygen therapy. Based on the study results, the authorized dose of REGEN–COV was reduced from 2400 to 1200 mg.Biological treatments of Crohn’s disease – efficacy compared head-to-head in a new studyIn a late-breaking abstract session at the Digestive Disease Week Virtual 2021 conference, which was held between 21 and 23 May, Janssen Pharmaceuticals made public new data on the safety and efficacy of Stelara® (ustekinumab) in the treatment of Crohn’s disease (CD) and ulcerative colitis (UC) [30]. Ustekinumab is a fully human monoclonal antibody already approved by the FDA for a number of indications including plaque psoriasis, psoriatic arthritis, CD and ulcerative colitis. It selectively inhibits both IL-12 and IL-23.The information presented included findings from the SEAVUE study, which compared ustekinumab versus adalimumab therapy in patients with moderate to severe active Crohn’s who had never previously been treated with antibody therapy for the disease. The study involved 386 patients who were randomized 1:1 to receive either approximately 6 mg/kg iv. at baseline followed by 90 mg sc. at 8-week intervals or initially adalimumab 160/80 mg by the sc. route and then 40 mg by the same route at 2-weekly intervals.The trial showed that high rates of disease remission were achieved, with 64.9% of participants treated with Stelara being in clinical remission as measured by the CD activity index at week 52. In addition, in excess of 55% patients were corticosteroid free and reported absence of symptoms and almost 30% were classed as being in endoscopic remission at the 1-year timepoint. However, ustekinumab treatment was not found to be statistically superior to adalimumab. The safety profiles were in line with that previously seen for both treatments, with discontinuation rates being slightly lower for the Stelara-treated cohort compared with the group receiving adalimumab (15.2 vs 23.6%).PatentsAquavit granted US patent protecting its novel microneedle technologyOn 4 May, Aquavit Pharmaceuticals, Inc. (NY, USA) issued a press statement regarding the granting of a patent by the United States Patent and Trademark Office (USPTO) giving IP protection to their AQT technology [31]. US Patent 10,980,865 has the title ‘Direct application system and method for the delivery of bioactive compositions and formulations’ and concerns a proprietary microneedle application system and method for the delivery of pharmaceuticals, vitamins and other molecules into the skin [32]. The delivery device has an exchangeable needle-head with a microneedle array. The needle head is attached to a reservoir containing the material to be delivered. During attachment the device is set to prevent leakage from the reservoir by a spring and plate mechanism. Delivery of the material in the reservoir is achieving by tapping the distal end of the device on the patient’s skin, thus allowing the microneedles to penetrate the skin and enabling flow from the reservoir through one or more multiple grooves in the microneedles. The spring and plate mechanism stops the flow on the completion of each tap.The company are exploiting their technology not only for cosmetic purposes, but for the delivery of drugs, biopharmaceuticals and vaccines, especially for conditions such as actinic keratosis and basal cell carcinoma where local delivery of therapeutics would minimize toxicity [33]. The patent also described the use of the technology in personalized medicine.European patent granted to Switzerland’s NLSMay brought good news for NLS Pharmaceutics Ltd (Stans, Switzerland) when the European Patent Office allowed patent number EP3426232, which provides intellectual property protection for the company’s lead product, Quilience® (mazindol CR) [34]. Quilience is under clinical development for the treatment of narcolepsy and has potential applications in other similar disorders [35]. The patent, which has the title ‘A Mazindol IR/SR multilayer tablet and its use for the treatment of attention deficit/hyperactivity disorder (ADHD)’ covers tablets comprising immediate-release and sustained-release layers of mazindol, and their use in patients suffering from attention deficit disorders or ADHD, alertness or vigilance deficit or conditions such as narcolepsy and idiopathic hypersomnia which result in daytime sleepiness [36].SummaryThis round-up of industry news covers May 2021. In many respects the most striking news came in the form of two FDA accelerated approvals for products targeted at specific mutations found in NSCLC and one EMA positive opinion for a gene therapy for a rare, progressive neurodegenerative disease in children. All three therapies offer hope to patients with previously very limited treatment options for significant improvements in prognosis and survival times. These therapies and their authorization underline how far drug discovery, development and regulatory science has progressed in exploiting the genetics underlying disease, biopharmaceutical manufacturing technology and new approaches to clinical development and regulatory review to bring products for unmet clinical needs rapidly to market.Financial & competing interests disclosureThe author has no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.No writing assistance was utilized in the production of this manuscript.
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